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Thank you for your interest in my blogs. Due to upcoming priorities, regretfully I will be discontinuing my writing.

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COVID news: SOTROVIMAB for emergency treatment, ACTEMRA for emergency use, Revoking EUA for non-NIOSH respirators, CARES Act for drug shortage mitigations, Updated vaccine risk information, EUA process infographic



INDICATION: Emergency use for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death

ADDRESSING UNMET NEED: Another option to help keep high-risk patients with COVID-19 out of the hospital

MECHANISM OF ACTION: Monoclonal antibody that is specifically directed against the spike protein of SARS-CoV-2 and is designed to block the virus’ attachment and entry into human cells


  • Phase 1/2/3 randomized, double-blind, placebo-controlled clinical trial, interim analysis, n= 583 non-hospitalized adults with mild-to-moderate COVID-19 symptoms and a positive SARS-CoV-2 test result
  • Primary endpoint: Progression of COVID-19 (defined as hospitalization for greater than 24 hours for acute management of any illness or death from any cause) through day 29
  • 1% with sotrovimab vs 7% with plaebo; 85% reduction
  • Ongoing monitoring of circulating viral variants and their sensitivity to sotrovimab
  • Laboratory testing showed retained activity against the current circulating variants reported in the UK, South Africa, Brazil, California, New York, India



ACTEMRA (tocilizumab)


INDICATION: Emergency use for treatment of hospitalized adults and pediatric patients (2 years of age and older) who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). Actemra is not authorized for use in outpatients with COVID-19.

ADDRESSING UNMET NEED: Additional therapeutic for those who do become hospitalized

MECHANISM OF ACTION: Monoclonal antibody that reduces inflammation by blocking the interleukin-6 receptor; in the case of COVID-19 infection, the immune system can become hyperactive, which may result in worsening of disease


  • Four clinical trials – randomized, controlled, open-label or double-blind placebo controlled, hospitalized patients with severe COVID-19 pneumonia
  • Primary endpoint: Death through 28 days of follow-up
  • Reduction is death with Actemra vs. usual care alone; also reduction in days to hospital discharge


  • Actemra is an FDA-approved prescription medication for multiple inflammatory diseases, including rheumatoid arthritis
  • EUA authorizes emergency use in certain hospitalized patients with COVID-19; not approved as a treatment for COVID-19.


Revoking EUA for Non-NIOSH-Approved or Decontaminated Disposable Respirators

 FDA is revoking EUAs for non-NIOSH-approved disposable respirators and the EUAs for decontamination and bioburden reduction systems

  • Crisis capacity conservation strategies were previously recommended to address respirator shortages earlier during the COVID-19 outbreak
  • Conservation strategies are no longer needed based on the increased domestic supply of respirators approved CDC and NIOSH
  • Health care personnel and facilities should only use FDA-cleared or NIOSH-approved respirators, including N95s
  • FDA has revoked EUAs for several non-NIOSH approved products


Coronavirus Aid, Relief, and Economic Security Act (CARES Act) Drug Shortage Mitigation Efforts

Coronavirus Aid, Relief, and Economic Security Act (CARES Act) was signed into law in 2020, to aid response efforts and ease the economic impact of COVID-19

  • Enhance ability to identify, prevent, and mitigate possible drug shortages and enhance visibility into drug supply chains
  • Agency works closely with drug manufacturers to prevent or reduce the impact of shortages

The specific authorities to enhance FDA’s ability to identify, prevent, and mitigate drug shortages are summarized below.

Specific authorities:

Updated Moderna and Pfizer-BioNTech Vaccine Risk Information

Suggested increased risks of myocarditis (inflammation of the heart muscle) and pericarditis (inflammation of the tissue surrounding the heart) following vaccination

  • Update follows an extensive review of information and the discussion by CDC’s Advisory Committee
  • Reports of adverse events suggest increased risks of myocarditis and pericarditis, particularly following the second dose and with onset of symptoms within a few days after vaccination
  • Updated Fact Sheets note that vaccine recipients should seek medical attention right away if they have chest pain, shortness of breath, or feelings of having a fast-beating, fluttering, or pounding heart after vaccination
  • More information being collected to assess longer-term outcomes

Moderna Fact Sheet

Pfizer-BioNTech Fact Shee

FDA’s EUA Process Infographic

EUA Process

Image credits: FDA

Drug Authorizations: ADUHELM for Alzheimer’s, AMONDYS 45 for DMD, TEMBEXA for Smallpox, WEGOVY for weight management, TRUSELTIQ for cholangiocarcinoma, KLOXXADO for opioid overdose



INDICATION: Treatment of Alzheimer’s disease

  • Accelerated approval based on reduction in amyloid beta plaques
  • Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s)

ADDRESSSING UNMET NEED: First novel therapy approved for Alzheimer’s disease since 2003; first to be directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain. Potential to address the gradual and cumulative devastation that Alzheimer’s disease causes, as patients lose their memory and cognitive functioning over time.

MECHANISM OF ACTION: Immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid beta; accumulation of amyloid beta plaques in the brain is a defining pathophysiological feature of Alzheimer’s disease,


  • Clinical trials for Aduhelm were the first to show that a reduction in beta amyloid plaques, a hallmark finding in the brain of patients with Alzheimer’s, and is expected to lead to a reduction in the clinical decline of dementia
  • Three double-blind, randomized, placebo-controlled dose-ranging studies, n-3,482 patients with Alzheimer’s disease
  • Significant dose-and time-dependent reduction of amyloid beta plaque with treatment


  • Warning: Amyloid-related imaging abnormalities, risk of hypersensitivity reactions, including angioedema and urticaria
  • Most common side effects: Headache, fall, diarrhea, and confusion/delirium/altered mental status/disorientation

REG PATHWAY: BLA, Accelerated approval

  • Based on surrogate endpoint (plaque reduction) that is expected to lead to a meaningful clinical benefit; required to conduct post-approval confirmatory studies to verify the anticipated clinical benefit
  • FDA offers perspective on the controversial approval; granted despite a negatve Advisory Committee vote



Sarepta Therapeutics

INDICATION: Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping

  • Approved under accelerated approval based on an increase in dystrophin production in skeletal muscle
  • Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials

ADDRESSING UNMET NEED: First targeted treatment for patients with this type of mutation; ~ 8% of DMD patients have a mutation that is amenable to exon 45 skipping

MECHANISM OF ACTION: Binds to exon 45 of dystrophin; allows production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping


  • Double-blind, placebo-controlled study, n=43 patients with genetically confirmed mutation of the DMD gene that is amenable to exon 45 skipping.
  • Significantly greater increase in dystrophin protein levels from baseline to week 48 with treatment vs placebo
  • Demonstration of increase in dystrophin production is reasonably likely to predict clinical benefit


  • Potential for kidney toxicity -observed in the nonclinical studies
  • Most common side effects: Upper respiratory tract infections, cough, fever, headache, joint pain and throat pain

REG PATHWAY: BLA, Accelerated approval


TEMBEXA (brincidofovir)


INDICATION FOR USE: Treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates

ADDRESSIGN UNMET NEED: Although naturally occurring smallpox no longer exists, concerns about potential uses of variola virus as a bioweapon has made smallpox drug development an important component of the U.S. medical countermeasures response.

MECHANISM OF ACTION: Antiviral drug against variola (smallpox) virus


  • Not been determined in humans because trials not feasible and inducing smallpox disease in humans is not ethical
  • Adequate and well-controlled animal efficacy studies of infected rabbits and mice
  • Statistically significant improvement in survival relative to placebo


  • Information clinical trials for a non-smallpox indication, primarily from patients who received hematopoietic stem cell transplants
  • Increased risk of death was seen in another disease (Cytomegalovirus disease – a viral infection) when Tembexa was used for a longer-than-recommended duration (longer than once a week for two weeks on days 1 and 8). Tembexa is only approved for the treatment of smallpox  
  • Most common side effects when using Tembexa are diarrhea, nausea, vomiting, and abdominal pain


  •  Approved under Animal Rule; allows findings from adequate and well-controlled animal efficacy studies to serve as the basis of an approval when it is not feasible or ethical to conduct efficacy trials in humans 
  • Priority ReviewFast Track and Orphan drug designations 
  • Developed in conjunction with the U.S. Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA)


FDA Smallpox preparedness

Wegovy (semaglutide)

Novo Nordisk

INDICATION : An adjunct to a reduced calorie diet and increased physical activity for chronic weight management in adults with an initial body mass index (BMI) of : 30 kg/m2 or greater (obesity) or 27 kg/m2 or greater (overweight) in the presence of at least one weight-related comorbid condition (e.g., hypertension, type 2 diabetes mellitus, or dyslipidemia)

Limitations of Use:
• Should not be coadministered with other semaglutide-containing products/ GLP-1 receptor agonist
• Safety and effectiveness in combination with other products intended for weight loss have not been established
• Not been studied in patients with a history of pancreatitis

ADDRESSING UNMET NEED: New drug treatment for chronic weight management, first since 2014

MECHANISM OF ACTION: GLP-1 receptor agonist that selectively binds to and activates the GLP-1 receptor, physiological regulator of appetite and caloric intake


  • Four randomized, double-blind, placebo-controlled trials, 68-week, n>2,600 on Wegovy vs n> 1,500 on placebo, average body weight 231 pounds (105 kg) and average BMI 38 kg/m2., 76% female
  • Average loss of initial body weight: 12.4% (no diabetes) and 6.2% (with diabetes) 


  • Boxed warning: Potential risk of thyroid C-cell tumors,
  • Should not be used in patients with a personal or family history of medullary thyroid carcinoma or in patients with a rare condition called Multiple Endocrine Neoplasia syndrome type 2 (MEN 2)
  • Most common side effects: Nausea, diarrhea, vomiting, constipation, abdominal (stomach) pain, headache, fatigue, dyspepsia (indigestion), dizziness, abdominal distension, eructation (belching), hypoglycemia (low blood sugar) in patients with type 2 diabetes, flatulence (gas buildup), gastroenteritis (an intestinal infection) and gastroesophageal reflux disease (a type of digestive disorder).  


  • Required pediatric assessments and Post-marketing commitments


TRUSELTIQ (infigratinib)

QED Therapeutics

FoundationOne® CDx

Foundation Medicine

INDICATION: Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test
This indication is approved under accelerated approval based on overall response rate and duration of response; continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

ADDRESSING UNMET NEED: Treatment option for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma 

MECHNISM OF ACTION: Small molecule kinase inhibitor of FGFR that supports proliferation and survival of malignant cells


  • Multicenter open-label single-arm trial, n=108 patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion determined by testing
  • Overall response rate (ORR) was 23% with 1 complete response and 24 partial responses
  • Median Duration of Response (DoR) was 5 months; 8 patients maintained response for 6 mo. or more    


  • Serious risks; Hyperphosphatemia and retinal pigment epithelial detachment
  • Most common adverse reactions: Hyperphosphatemia, increased creatinine, nail toxicity, stomatitis, dry eye, fatigue, alopecia, palmar-plantar erythrodysesthesia syndrome, arthralgia, dysgeusia, constipation, abdominal pain, dry mouth, eyelash changes, diarrhea, dry skin, decreased appetite, vision blurred and vomiting

REGULATORY PATHWAY: NDA, Accelerated Approval

  • Review conducted under Project Orbis, for concurrent submission and review of oncology drugs among international partners
  • FDA collaboration with the Australian Therapeutic Goods Administration (TGA) and Health Canada
  • Used the Real-Time Oncology Review (RTOR) pilot program for streamlined data submission and Assessment Aid and Product Quality Assessment Aid (PQAA)
  • Granted priority review, fast-track designation, and orphan drug designation


KLOXXADO (naloxone hydrochloride)


INDICATION: For the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and /or central nervous system depression, for adult and pediatric patients
-Is intended for immediate administration as emergency therapy in settings where opioids may be present
-Is not a substitute for emergency medical care

ADDRESSIBNG UNMET NEED:  Higher dose naloxone hydrochloride nasal spray for combatting opioid overdose

MECHANISM OF ACTION: Antagonizes opioid effects by competing for the same receptor sites; reverses effects of opioids, including respiratory depression, sedation and hypotension


  • Scientific justification based on FDA’s finding of safety and effectiveness for naloxone hydrochloride (NARCAN injection)
  • Specific pharmacokinetic data to establish the drug’s safety and efficacy for its approved use


  •  New product delivers 8 mg naloxone into nasal cavity; FDA had previously approved 2 mg and 4 mg naloxone nasal spray products



Image credits: Biogen, Sarepta, Chimerix, Novo Nordisk, QED, Hikma

News and Views: 2022 budget, Enhance Equity initiative, Enhance drug supply chain, Reg Science focus areas, OSEL updates, CURE ID platform expansion, Cosmetics regulation

2022 Budget for Public Health Infrastructure and Product Safety Programs

Requesting a total budget of $6.5 billion as part of the President’s fiscal year (FY) 2022 budget

  • $185 million in additional investments in FDA’s Critical Public Health Infrastructure
  • $97 million in additional investments in Core FDA Safety Programs
  • $61 million in additional investments in Public Health Issues Confronting the U.S.


Enhance Equity Initiative

Enhance Equity Initiative to promote and protect the health of racial and ethnic minorities, underrepresented and underserved populations through research and communication strategies that address health disparities


Enhance US Drug Supply Chain

Efforts to enhance supply chain to ensure quality of prescription drugs and safeguard integrity of pharmaceutical distribution 

Key Focus Areas for Regulatory Science Investment and Innovation

Office of Regulatory Science and Innovation (ORSI) supports cross-agency collaborative programs aligned with FDA’s strategic priorities for advancing regulatory science

  1. Increasing Choice and Competition through Innovation 
  2. Unleashing the Power of Data
  3. Empowering Patients and Consumers
  4. Public Health Emergency Preparedness and Response

The fourth was added post COVID experience.


Office of Science and Engineering Laboratories (OSEL) updates


HHS grant to expand CURE ID platform for COVID-19 treatments

$9.2 million grant for expansion of the CURE ID platform for COVID-19

  • CURE ID allows global clinical community to share novel uses of existing drugs for challenging infectious diseases
  • New expansion will allow automated data collection from electronic health records (EHR) worldwide and clinical disease registries for COVID-19
  • Will enable health care providers and researchers access to comprehensive (de-identified) case reports on tens of thousands of patients, including treatment outcomes, such as recovery, deterioration, hospitalization, ICU admission, and death


Cosmetics Are Not FDA-Approved, but Are FDA-Regulated

Cosmetic definition: Intended to be rubbed, poured, sprinkled, or sprayed on, introduced into, or otherwise applied to the human body…for cleansing, beautifying, promoting attractiveness, or altering the appearance

  • does not require cosmetic products and ingredients, other than color additives, to have FDA approval
  • must ensure safety of product
  • laws and regulations for interstate commerce e.g. prohibition of adulterated or misbranded cosmetics in interstate commerce
  • cannot make therapeutic claims such as for treating or preventing disease, or to affect the structure or function of the body


Image credit: FDA

DEVICE AUTHORIZATIONS: TALON SPACER for avascular necrosis, GILI biosensor for touchless heart and respiratory rates, GI GENIUS for colonic mucosal lesions, IPSIHAND for stroke rehabilitation

Patient Specific Talus Spacer

 Additive Orthopaedics

INDICATION FOR USE: For avascular necrosis of the ankle joint. The anatomical landmarks necessary for the design and creation of the Additive Orthopaedics Patient Specific Talus Spacer must be present and identifiable on computed tomography scan.

ADDRESSING UNMET NEED: Avascular necrosis is a rare, serious and potentially debilitating that can cause removal of part of leg; provides patients with treatment option that could potentially reduce pain, retain range of motion of joint and improve quality of life

DECRIPTION: Additively manufactured implant made from cobalt chromium metal alloy and produced by laser sintering

  • Made for each patient individually, modeled from computed tomography (CT) imaging, and is fitted to a patient’s specific anatomy
  • During replacement surgery, patient’s talus bone is removed and replaced with implant made from cobalt chromium alloy
  • Intended to be a joint-sparing procedure, as it allows the patient to retain motion in the ankle joint


  • Results from 31 patients and 32 talus replacement surgeries (one patient had operations on both ankles) with the implant
  • Probable benefit endpoint: Reduction in baseline level pain following surgery using the Visual Analog Scale (“VAS”) + ankle range of motion (“ROM”), Foot and Ankle Outcome Scores (“FAOS”) and FAOS subscales on activities of daily living (“ADL”), ability to perform sports and recreational activities, QoL
  • Primary safety endpoint: Secondary subsequent surgical intervention (“SSSI”) + adverse events
  • At three years post-operation, the average reported pain decreased from “moderate to severe” prior to surgery to “mild” post-surgery, and average range of motion in the ankle joint also improved
  • By the three-year mark, 3 reported additional surgeries
  • The most common reported adverse events were pain and scar tissue at the surgery site

REGULATORY PATHWAY: Humanitarian Device Exemption

  • Trade/Device Name: Patient Specific Talus Spacer
  • Product Code: QNN
  • Annual Report requirements
  • Post-approval study: Patient Specific Talus Spacer Registry PAS is a prospective post-approval US registry study to provide ongoing safety and probable benefit assessment


Gili Pro BioSensor

ContinUse Biometrics Ltd.

INDICATION FOR USE: Includes an optical module that is intended to capture motion-vibration signals from an illuminated surface for assessment of physiological information. Such information, captured during spot-measurement, includes: Heart rate, Respiratory rate

The device is indicated for use by or under the supervision of healthcare professionals for adult patients in a hospital, outpatient, or other medical care settings, or for clinical research purposes. The device should be used while the subject is seated upright either in a chair or in a bed. The information stored on the system may be reviewed by qualified persons.

DEVICE TYPE: Hardware and software for optical camera-based measurement of pulse rate, heart rate, breathing rate, and/or respiratory rate.

  • The device uses an optical sensor system and software algorithms to obtain and analyze video signal and estimate pulse rate, heart rate, respiratory rate and/or breathing rates.
  • Not intended to independently direct therapy


  • Representative of the intended use population for the device
  • Demonstrate output consistency using the expected range of data sources and data quality encountered in the intended use population and environment
  • Compare device output with a clinically accurate patient-contacting relevant comparator device in an accurate and reproducible manner


  • Delayed or incorrect treatment due to erroneous output as a result of device malfunction or algorithm error
  • Delayed or incorrect treatment due to user misinterpretation
  • Eye damage, burns, and related safety concerns due to illuminating optics


  • Regulation Number: 21 CFR 870.2790
  • Regulation Name: Hardware and software for optical camera-based measurement of pulse rate, heart rate, breathing rate, and/or respiratory rate
  • Regulatory Class: Class II
  • Product Code: QOK


GI Genius

Cosmo Artificial Intelligence – AI, LTD /Medtronic

INDICATION FOR USE: Computer-assisted reading tool designed to aid endoscopists in detecting colonic mucosal lesions (such as polyps and adenomas) in real time during standard white -light endoscopy examinations of patients undergoing screening and surveillance endoscopic mucosal evaluations. The GI Genius computer-assisted detection device is limited for use with standard whitelight endoscopy imaging only. This device is not intended to replace clinical decision making.

DEVICE TYPE: Gastrointestinal lesion software detection system

  • Computer-assisted detection device used in conjunction with endoscopy for the detection of abnormal lesions in the gastrointestinal tract. This device with advanced software algorithms brings attention to images to aid in the detection of lesions. The device may contain hardware to support interfacing with an endoscope


  •  Demonstrate that device performs as intended under anticipated conditions of use, including detection of gastrointestinal lesions and evaluation of all adverse events


  • Algorithm failure leading to – False positives resulting in unnecessary patient treatment; or False negatives resulting in delayed patient treatment
  • Failure to identify lesions, resulting in delayed patient treatment, due to software/hardware failure including: Incompatibility with hardware and/or data source, Inadequate mapping of software architecture, Degradation of image quality, Prolonged delay of real-time endoscopic video


  • Regulation Number: 21 CFR 876.1520
  • Regulation Name: Gastrointestinal lesion software detection system
  • Regulatory Class: II
  • Product Code: QNP


IpsiHand Upper Extremity Rehabilitation System

Neurolutions Inc

INDICATION FOR USE: For use in chronic stroke patients (≥ 6 months post-stroke) age 18 or older undergoing stroke rehabilitation, to facilitate muscle re-education and for maintaining or increasing range of motion in the upper extremity.

DEVICE TYPE: Electroencephalography (EEG)-driven upper extremity powered exerciser

  • An EEG-driven upper extremity powered exerciser is a non-invasive prescription device intended for rehabilitation by driving movement or exercise of an impaired upper extremity in response to the detection of purpose oriented electrical activity produced by the patient’s brain


  • Unblinded study of 40 patients over a 12-week trial
  • All participants demonstrated motor function improvement with the device over the trial
  • Adverse events reported included minor fatigue and discomfort and temporary skin redness 


  • Device provides ineffective treatment, leading to worsening condition
  • Unintended motion leading to injury
  • Thermal injury including burns and shock
  • Adverse tissue reaction
  • Cross contamination, leading to infection or adverse tissue reaction
  • Pain or discomfort including: Headache, Fatigue, Skin redness

REGULATORY PATHWAY: De Novo request, Breakthrough designation

  • Regulation Number: 21 CFR 890.5420
  • Regulation Name: Electroencephalography (EEG)-driven upper extremity powered exerciser
  • Regulatory Class: Class II
  • Product Code: QOL


Image credit: Respective device manufacturers

News and Views: Janssen COVID vaccine pause lifted, Guidance for developing COVID drugs, New pharmacometrics approach, Standards to ban menthol cigarettes, CDRH patient science, Opioid safe disposal, Colorectal cancer screening

FDA and CDC Lift Recommended Pause on Janssen Vaccine Use Following Thorough Safety Review

Based on confidence in vaccine’s safety and effectiveness following data assessment

  • Vaccine’s known and potential benefits outweigh its known and potential risks in individuals 18 years of age and older
  • Chance of clots is very low, but the FDA and CDC will remain vigilant in continuing to investigate this risk
  • Fact Sheet for Recipients and Caregivers, provide information on risk


COVID-19: Developing Drugs and Biological
Products for Treatment or Prevention

Guidance describes current recommendations regarding phase 2 and phase 3 trials for drugs under development to treat or prevent COVID-19

  • Population
  • Trial Design and Conduct
  • Efficacy Endpoints
  • Safety Considerations
  • Statistical Considerations


A New Approach to Pharmacometrics: Recurrent Neural Networks for Modeling Drug Exposure and Drug Response

Pharmacometric models based on recurrent neural network to simulate the time course of pharmacodynamic (PD) response that develops latently, according to complex biological intermediate step

  • A long short-term memory recurrent neural network (LSTM RNN) trained to use time sequence of simulated plasma concentrations along with patient baseline PD values and patient demographics to predict PD response in single dose regimen
  • Machine Learning model able to accurately predict the PD response of individuals who were treated with different dosing regimens
  • LSTM RNNs may complement traditional PK/PD models in the area of highly complex PK/PD data analysis and possibly facilitate development of predictive models with improved accuracy.  


Efforts to ban menthol cigarettes to reduce addiction and youth experimentation, improve quitting, and address health disparities

Two tobacco product standards within the next year to ban menthol as a characterizing flavor in cigarettes and ban all characterizing flavors (including menthol) in cigars

  • Menthol masks unpleasant flavors and harshness of tobacco products, more addictive and harder to quit
  • Banning menthol cigarettes would lead an additional 923,000 smokers to quit, including 230,000 African Americans, avert 633,000 deaths including about 237,000 for African Americans


CDRH Patient Science and Engagement

CDRH puts patients first with its Patient Science and Engagement Program

  • Communicating COVID-19 testing to underrepresented populations
  • Gender differences for heart failure Patient-Reported Outcome measure
  • Ranking factors influencing women’s decision making on uterine fibroids surgery decision making
  • Workshop of Patient-Generated Health Data and its use across the healthcare ecosystem and during medical device development, evaluation, and monitoring


Critical Importance of Safe Disposal of Opioids  

Unused or expired medicines in the home have long been a major safety concern and implicated in most some common and most severe cases of accidental ingestion by children

  • Removing prescription opioids once no longer medically necessary can prevent accidental exposure or intentional misuse
  • Access drug take back site, check if a drug is on the  “flush list
  • FDA exercises oversight on packaging and disposal of drugs that pose risks of abuse or overdose, such as opioid analgesics
  • Take advantage of the National Prescription Drug Take Back Day 

Where and How to Dispose

Colorectal Cancer Screening Options

The only way to find a polyp, precancerous growth, or colorectal cancer is to do a screening test or procedure

  • FDA authorized Colorectal Cancer Screening: Stool sample tests (at-home stool test) and Visual screening procedures (colonoscopy, Flexible Sigmoidoscopy, CT Colonography)
  • Some Minority Groups Are at Higher Risk: Black men and women as well as American Indian and Alaska Native people
  • Colorectal Cancer Rates Are Up Among Adults Under 50


Image credit: FDA, CDC

COVID-19 News: Janssen vaccine pause Q/A, Vaccine quality, #VaccineReady campaign, 2020 CDRH innovation, Bamlanivimab EUA revoked, Pooling and serial testing of diagnostics, Remote interactive inspections

J&J Vaccine Pause Q&A

The FDA and CDC are reviewing data involving cases of a low level of platelets in combination with a rare and severe type of blood clot – cerebral venous sinus thrombosis (CVST)

  • Out of an abundance of caution, the FDA and CDC are recommending a pause in J&J vaccines
  • The agencies are investigating all reports to determine a causal relationship
  • The agencies are ensuring health care providers for proper symptom recognition and management due to the unique treatment required for this condition
  • For more information, visit this CDC page

Important Steps to Ensure Quality, Safety and Effectiveness of Authorized COVID-19 Vaccines

FDA’s inspections review the quality of manufacturing procedures, including records, staff training, facility operations, drug production and testing and the systems in place to ensure product quality

  • During an inspection of Emergent BioSolutions, production site for J&J vaccine, a number of concerning observations (Form 483)
  • Pause new vaccine production while issues are resolved
  • For vaccines already manufactured, additional testing before distribution

Emergent Form 483

#VaccineReady campaign

FDA’s Office of Minority Health and Health Equity has joined the U.S. DHHS #VaccineReady campaign to address vaccine disparities

  • Developed social medial messages to encourage vaccination of diverse communities
  • Twitter and Facebook messages with a variety of graphics and videos on topics such as vaccine development, vaccine authorization, and the benefits of getting vaccinated against COVID-19

    TOOLKIT download and share

    Twitter Graphics: Download and Share    

   Facebook Graphics: Download and Share

2020 CDRH innovation to combat pandemic

High volume of EUAs including those for novel devices

Remarkable ability of the device ecosystem and the FDA to adapt and respond to emerging public health needs

Bamlanivimab EUA revoked

Revoked EUA for bamlanivimab, when administered alone, for treatment of mild-to-moderate COVID-19 in adults and certain pediatric patients 

  • Ongoing analysis of emerging scientific data, specifically the sustained increase of variants resistant to bamlanivimab alone
  • Other monoclonal antibody therapies authorized for EUA remain appropriate treatment choices


Streamlining Pooling and Serial Testing for Certain Molecular Diagnostic Tests

Amendment to allow certain authorized molecular diagnostic tests to be distributed and used to pool anterior nasal respiratory specimens from asymptomatic individuals

  • Prior to a test being distributed or used for any new indication, the developer must submit a notification to the FDA with the information required by the amendment


Remote Interactive Inspections of Drug Manufacturing and Bioresearch Monitoring Facilities

Use of various interactive and virtual tools for remote interactive evaluation of manufacturing facilities

  • Will use existing risk management methods and related tools to determine remote interactive evaluation
  • For pre-approval and pre-license, post-approval, surveillance, for-cause and bioresearch monitoring programs 


Image credit: FDA. CDC. Emergent, Lilly

COVID-19 News: FDA-CDC statement on J&J vaccine, SYMBIOTICA antibody test, PREPP plan update, Addressing health disparities, OTC and POC Tests

Joint CDC and FDA Statement on Johnson & Johnson COVID-19 Vaccine

CDC and FDA are reviewing data involving six reported U.S. cases, out of 6.8 M doses administered, of a rare and severe type of blood clot in individuals after receiving the J&J vaccine

  • Blood clot called cerebral venous sinus thrombosis (CVST) seen in combination with low levels of blood platelets (thrombocytopenia)
  • Occurred among women between the ages of 18 and 48, and symptoms occurred 6 to 13 days after vaccination
  • Heparin, commonly used for clots, should not be used this type of clot

Advisory Committee on Immunization Practices (ACIP) on 4/14 to further review these cases and assess potential significance

  • Recommending pause in vaccine use out of an abundance of caution
  • Health care providers are asked to report adverse events to the Vaccine Adverse Event Reporting System at


SYMBIOTICA COVID-19 Self-Collected Antibody Test System


INDICATION: Qualitative detection of IgG antibodies to SARS-CoV-2 in human fingerstick blood dried blood spot (DBS) specimens that are self-collected at home by an individual age 18 years or older or
collected by an adult from an individual 5 years of age and older using the COVID-19 Self-Collected Antibody Test System Collection Kit

Intended for use as an aid in identifying individuals with an adaptive immune response to SARS-CoV-2, indicating recent or prior infection

ADDRESSING UNMET NEED: First prescription use, home collection antibody test. Important in identifying individuals who have developed an adaptive immune response from a recent or prior COVID-19 infection


  • Enzyme-linked immunosorbent assay (ELISA) for the qualitative detection of IgG antibodies to SARS-CoV-2 in human fingerstick dried blood spot (DBS) specimens
  • COVID-19 Self-Collected Antibody Test System Collection Kit for home collection
  • eSTAD ELISA assay used in combination with Calibrator (IgG, Human), Positive control and Negative control

REGULATORY PATHWAY: Emergency Use Authorization

Letter of Authorization

Update on COVID-19 Pandemic Recovery and Preparedness Plan (PREPP) Initiative

An independent committee PREPP report identified a number of options for strengthening FDA’s COVID-19 response; five areas have been identified for initial focus

  • Improving EUA process
  • Timely and accurate information to public
  • Further inspectional approaches by using next-gen technologies
  • Improve supply chain resilience


Vaccine Ready: Addressing COVID-19 Health Disparities among Racial and Ethnic Minority Communities

All authorized vaccines tested in clinical trials including participants from racial and ethnic minority, American Indian and Alaska Native, and other diverse communities

  • FDA review adheres to rigorous standards for safety and effectiveness
  • Working working hand-in-hand with the FDA’s Office of Minority Health and Health Equity (OMHHE) to address concerns about vaccines among diverse communities.


Advancement of Over-the Counter and Other Screening Test Development

Point of Care (POC) testing and Over-the-Counter (OTC) tests important part of pandemic response

  • Quidel QuickVue At-Home OTC COVID-19 test – authorized for OTC at-home serial screening
  • Abbott BinaxNOW (multiple configurations)
    • Abbott BinaxNOW COVID-19 Antigen Self Test – authorized for OTC at-home serial screening
    • Abbott BinaxNOW COVID-19 Ag Card 2 Home Test – authorized for OTC at-home serial screening with telehealth proctor
    • Abbott BinaxNOW COVID-19 Ag 2 Card – authorized for POC serial screening without a prescription 
  • BD Veritor System for Rapid Detection of SARS-CoV-2 – authorized for POC serial screening with a prescription

These authorizations based on efforts to streamline the process for test developers and information to help schools, workplaces, communities, and others establish screening programs. 


Image credit: FDA

Marketing Authorizations: ABECMA for multiple myeloma, PoNS for gait in multiple sclerosis, HARMONY for pulmonary valve regurgitation, Q-COLLAR for brain injury protection, ClearUP for sinus pain

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ABECMA (idecabtagene vicleucel) injection

Bristol-Myers Squibb

INDICATION: B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an antiCD38 monoclonal antibody

USE: Each dose of Abecma is a customized treatment created by using a patient’s own T-cells which are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells. Once the cells are modified, they are infused back into the patient. 

ADDRESSING UNMET NEED: First cell-based gene therapy for adult patients with multiple myeloma

MECHANISM OF ACTION: Chimeric antigen receptor (CAR)-positive T cell therapy targeting B-cell maturation antigen (BCMA), expressed on normal and malignant plasma cell surfaces. Antigen-specific activation of ABECMA results in CAR-positive T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells


  • Open-label, single-arm, multicenter study in adults (n=127) with relapsed and refractory multiple myeloma who had received at least 3 prior lines of antimyeloma therapy
  • Endpoints: Overall response rate (ORR), stringent complete response (sCR) rate, Very good partial response (VGPR), duration of response (DOR) based on the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma
  • ORR=72%, sCR=28%, VGPR=25%, PR=19%, median DOR (PR or better)= 11 mo.


  • Boxed warning:  Cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), neurologic toxicity, and prolonged cytopenia, all of which can be fatal or life-threatening.
  • Most common side effects: CRS, infections, fatigue, musculoskeletal pain, and a weakened immune system
  • REMS: Certified staff involved in the prescribing, dispensing or administering of Abecma trained to recognize and manage CRS and nervous system toxicities and other side effects


  • Orphan Drug and Breakthrough Therapy designations
  • Post-marketing observational safety study involving patients treated with Abecma


Portable Neuromodulation Stimulator (PoNS)

Helius Medical

INDICATION FOR USE: Short-term treatment of gait deficit due to mild to moderate symptoms from multiple sclerosis

ADDRESSING UNMET NEED: New aid in physical therapy for those who live with MS on a daily basis

DESCRIPTION: Portable, non-implantable delivery of mild neuromuscular electrical stimulation to dorsal surface of tongue

  • Consists of controller and mouthpiece connected to each other by a cord
  • Mouthpiece is held lightly in place by the lips and teeth and control unit is worn around the neck
  • Controller sends signals to mouthpiece; receptors on tongue send millions of neural impulses to the brain
  • Additionally, therapist can connect the control unit to a computer and view usage data to improve a patient’s therapy by identifying potential areas of missed or shortened sessions


  • Two clinical studies and a retrospective analysis of real-world data (RWD)
  • First Study: Randomized, double blind controlled trial (n=20 MS patients), PoNS vs sham control : Dynamic Gait Index (DGI) at 2, 6, 10, 14 weeks showed statistically and clinically significant imporvement with PoNS
  • Second Study: Randomized controlled trial (n=14 MS patients). PoNS vs sham control : Effect on cognitive and physical rehab. Sensory Organization Tasks (SOT) and DGI scores at 14 weeks showed significant improvement
  • RWD study: Retrospective analysis of patient feedback Functional Gait Assessment in clinical rehabilitation settings
  • Safety: Prescription use only in accordance with warnings and precautions (inducing electrical safety) in labeling

REGULATORY PATHWAY: De Novo, Breakthrough designation

Product Code QCF

HARMONY Transcatheter Pulmonary Valve (TPV) System


INDICATION FOR USE: To improve blood flow to the lungs in patients with severe pulmonary valve regurgitation without open-heart surgery, which is the current standard of care. Patients have a native or surgically-repaired right ventricular outflow tract (RVOT). The use of the Harmony valve may delay the time before a patient needs additional open-heart surgery. It can also potentially reduce the total number of open-heart surgeries required over an individual’s lifetime.

ADDRESSING UNMET NEED: New less-invasive treatment alternative to open-heart surgery for adult and pediatric patients with certain types of congenital heart disease. May help patients improve their quality of life and return to their normal activities more quickly.

DESCRIPTION: Pulmonary Valve Prosthesis percutaneously delivered

  • Intended to replace patient’s pulmonary heart valve; different from heart valves in that they are placed percutaneously and do not require open chest surgery or a cardiotomy for placement
  • During the implantation procedure, a thin, hollow tube (catheter) with a collapsed Harmony valve on the end is inserted through a vein in the groin or in the neck and into the right side of the heart, and then into the RVOT where it is placed into position
  • Valve is then released from the catheter; it expands on its own, and anchors to the RVOT
  • Once new valve is in place, it opens and closes like a door to force the blood to flow in the correct direction


  • Prospective, non-randomized, multi-center clinical study, n=70 patients
  • Follow-up examinations at study start, at implant procedure, discharge, and post implant at one month, six months, and annually through five years
  • Primary effectiveness endpoint: 89.2% patients had no additional surgical or interventional procedures related to the device and acceptable heart blood flow function at six month
  • Primary safety endpoint: No procedure- or device-related death within 30 days following implant
  • Adverse events: Irregular or abnormal heart rhythm, ventricular tachycardia), leakage around the valve, minor bleeding, narrowing of the pulmonary valve, movement of implant


  • Breakthrough Device Designation 
  • Part of the U.S.-Japan Medical Device Harmonization by Doing Collaboration, to promote timely access to innovative devices in both the U.S. and Japan
  • Post-approval study with 10 years followup

Product Code NPV

Q30 Sports Science

INDICATION FOR USE: Non-invasive device intended to be worn around the neck of athletes aged 13 years and older during sports activities to aid in the protection of the brain from effects associated with repetitive sub-concussive head impacts. For OTC use.

ADDRESSING UNMET NEED: Protective equipment for athletes when playing sports to help protect their brains from the effects of repetitive head impacts


  • C-shaped collar that provides compressive force to the internal jugular veins, which in turn increases the blood volume in the skull’s blood vessels
  • Blunt trauma accidents cause brain moves unrestrained in the skull – “slosh”
  • Q-Collar’s increase in blood volume in those blood vessels creates a tighter fit of the brain inside the skull and reduces the “slosh” movement
  • By reducing brain movement within cranial space, Q-Collar may aid in protection from effects of head impacts


  • Prospective, longitudinal study, n=284 subjects 13 years or older from a high school football team, Q-collar vs no collar; all wore accelerometer device that measured every head impact during play
  • Diffusion Tensor Imaging (MRI type) of brain to compare structural changes pre-season and post-season
  • Significant deep tissues changes (involved in transmission of electrical nerve signals) in 73% no-Collar group vs. 13% in Q-collar group
  • No significant adverse events with device use

REGULATORY PATHWAY: De Novo classification


ClearUP Sinus Relief

Tivic Health Systems

INDICATION FOR USE: Transcutaneous electrical nerve stimulator that electrically stimulates the skin overlying the paranasal sinuses and is intended to be used for the temporary relief of moderate to severe congestion. ClearUP Sinus Relief is a treatment to be used at home by individuals 18 and older

ADDRESSING UNMET NEED: OTC availability of device for sinus pain treatment

DESCRIPTION: Transcutaneous electrical nerve stimulator for the relief of congestion.

  • Electrically stimulates the skin overlying the paranasal sinuses to relieve congestion

REGULATORY PATHWAY: De Novo reclassification


Image credit: Bristol-Myers Squibb, Helius Medical, Medtronic, Q30 Sports, Tivic Health

COVID-19 News: COVID Plus monitor, BioFire respiratory panel, Screening vs Diagnostic Testing, Cure ID App


Tiger Tech Solutions, Inc.

INDICATION FOR USE: Non-diagnostic screening device that identifies certain biomarkers that are indicative of some types of conditions, such as hypercoagulation.

ADDRESSING UNMET NEED: First Machine Learning-Based screening device to identify certain biomarkers that may indicate COVID-19 infection. Combining use of this new screening device with temperature checks could help identify asymptomatic individuals.


  • Arm-band with two embedded photoplethysmography (PPG) sensors and a processor that is wrapped around the recipient’s bare left arm above the elbow
  • PPG sensors acquire direct pulsatile biosignals over 3-5 minutes; processor interprets signals
  • Fed into a probabilistic machine learning model trained to make predictions on whether the individual is showing certain signals, such as hypercoagulation
  • Green light (not demonstrating biomarkers), red light (demonstrating biomarkers), blue light (test is inconclusive) due to indeterminate features


  • Validation study : Hospital study (n=467 asymptomatic individuals)
  • Positive percent agreement (proportion of the COVID-19 positive individuals identified correctly by the device to possess certain biomarkers) of 98.6%
  • Negative percent agreement (proportion of the COVID-19 negative individuals identified correctly by the device to not possess certain biomarkers) of 94.5%
  • Confirmatory study: School study, with similar performance

REGULATORY PATHWAY: Emergency Use Authorization

EUA Authorization Letter

BioFire Respiratory Panel

Biofire Diagnostics

INDICATION FOR USE: PCR-based multiplexed nucleic acid test intended for use with the BioFire FilmArray 2.0 or BioFire FilmArray Torch systems for the simultaneous qualitative detection and identification of multiple respiratory viral and bacterial nucleic acids in nasopharyngeal swabs (NPS) obtained from individuals suspected of respiratory tract infections, including COVID-19.

ADDRESSING UNMET NEED: Multi-analyte molecular test that simultaneously detects 22 different viruses and bacteria associated with respiratory tract infections, including SARS-CoV-2, from a single nasopharyngeal swab.

DEVICE TYPE: Device to detect and identify nucleic acid targets in respiratory specimens from microbial agents that cause the SARS-CoV-2 respiratory infection and other microbial agents when in a multi-target test.

Is for use only for people suspected of respiratory tract infections, including COVID-19. 

Should be used by health care providers in combination with a clinical examination and consideration of patient medical history.

REGULATORY PATHWAY: First SARS-CoV-2 Diagnostic Test Using Traditional Premarket Review – De Novo pathway

  • Regulation Number: 21 CFR 866.3981
  • Regulatory Class: Class II
  • Product Code: QOF


Screening vs Diagnostic Testing

Diagnostic testing: Identifies current infection at the individual level and is performed when a person has signs or symptoms of infection, or when a person is asymptomatic but has recent known or suspected exposure.

Screening testing: Looks for individual infections in a group even if there is no reason to, Testing asymptomatic individuals who do not have known or suspected exposure to COVID-19 in order to make individual decisions based on the test results.

Test developers decide the use they will seek to have authorized by the FDA.

The FDA issued a new supplemental template for test developers seeking EUA of certain tests for screening with serial testing.


CURE ID App receives award

 CURE ID is an internet-based repository for clinical community report novel uses of existing drugs for difficult-to-treat infectious diseases

  • Collaboration between FDA, National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), and World Health Organization (WHO)
  • Critical updates to app to allow clinicians to more easily report their real-world experiences treating COVID-19 patients who are unable to be enrolled in a clinical trial
  • Received 2021 Golan Christie Taglia Patient Impact Philanthropy Award


Image credit: FDA, Tiger Tech, BioFire

News and Views: Fraudulent FDA ‘registered’ products, Reporting misconduct, Data modernization plan, Real World Evidence for regulatory decision making

FDA Registered” or “FDA Certified” Medical Devices are not FDA approved

Misleading website claims of FDA Registered, FDA Certified, FDA Registration Certificate aometimes along with FDA logo

  • Manufacturers register annually with the FDA for the production and distribution of medical devices intended for use in the US
  • However, entry in the FDA’s registration and listing database does not denote approval, clearance
  • Also,  FDA does not issue any type of device registration certificates to medical device facilities.
  • Firms misleadingly displaying certificates to imply review or approval by FDA are misbranding’ the product and are in violation of the Federal Food, Drug, and Cosmetic Act
  • Check for Approved and Cleared Products in the Devices@FDA Database


Reporting Allegations of Regulatory Misconduct

Allegation of regulatory misconduct is a claim that a medical device manufacturer or individuals marketing medical devices may be doing so in a manner that violates the law

  • Make the FDA aware of regulatory concerns it may not learn of otherwise
  • Help the FDA identify the potential risks to patients

Examples include: False promotion or advertising of a device, not submitting required safety reports, noncompliant manufacturing practices, failing to register/list product, not meeting import requirements, document falisfication etc.

Submit an allegation through the Allegations of Regulatory Misconduct Form


Data Modernization Action Plan for Putting Data to Work for Public Health

Data Modernization Action Plan (DMAP) to modernize FDA’s technical infrastructure

  1. Identify and execute high value driver projects for individual centers and for the Agency;
  2. Develop consistent and repeatable data practices across the Agency; and,
  3. Create and sustain a strong talent network combining internal strengths with key external partnerships.

DMAP driver projects will contribute to the FDA’s public health responsibilities in the near term while also building critical capabilities for the future.  


Graphic with the words Real World Data and Real World Evidence and the acronyms RWD and RWE and icons of a stethoscope, beakers, computer monitor and a heart

Leveraging Real World Evidence in Regulatory Submissions of Medical Devices

Real World Evidence (RWE) is the clinical evidence regarding the usage, and benefits and risks, of a medical product derived from the analysis of Real World Data (RWD)


Image credit: FDA

COVID-19 News: Cue OTC test, EUA Public FAERS dashboard, Roche false results, Ivermectin warning, Learn about treatment options

CUE Test for Home and Over The Counter (OTC) Use

Cue Health

INDICATION FOR USE: Qualitative detection of nucleic acid from SARS-CoV-2 in anterior nasal (nasal) swab specimens collected with the Cue Sample Wand.

Intended for use in adults (self-swabbing) or children ≥2 years of age (swabbed by an adult) with or without symptoms or other epidemiological reasons to suspect COVID-19. For Home and OTC use
authorized for non-prescription home use.

ADDRESSING UNMET NEED: First molecular OTC diagnostic test to continue to expand access to testing to help us in the fight against the pandemic


  • For qualitative detection of nucleic acid from SARS-CoV-2 in anterior nasal (nasal) swab specimens collected with the Cue Sample Wand
  • Test includes: the single-use Cue COVID-19 Test Cartridge, the single-use Cue Sample Wand nasal swab, the Cue Cartridge Reader the Cue Health Mobile Application (App)
  • Battery-operated Cue Cartridge Reader runs the Cue Test Cartridge and communicates results directly to the Cue Health App in about 20 minutes


  • Correctly identified 96% of positive samples from individuals known to have symptoms
  • Correctly identified 100% of positive samples from individuals without symptoms



New COVID-19 EUA FAERS Public Dashboard 

Launch of FDA Adverse Event Reporting System (FAERS) Public Dashboard to access data on adverse events

  • User-friendly search tool for adverse events associated with the use of drugs and biologics
  • Provides weekly updates of adverse event reports and increases transparency and provide more efficient access to safety information.


Potential for False Results with Roche Molecular Systems, Inc. cobas SARS-CoV-2 & Influenza Test

False positive results can occur with the Roche Molecular Systems, Inc. (Roche) cobas SARS-CoV-2 & Influenza A/B Nucleic Acid Test for use on the cobas Liat System

  • Assay tubes may sporadically leak, causing an obstructed optical path in the Liat analyzer, producing abnormal PCR growth curves
  • Abnormal PCR cycling in the reaction tubes may also produce abnormal PCR growth curves, leading to erroneous results


  • Monitor for unexpected clusters of positive Flu B results
  • Repeat tests when two or three analytes are positive
  • Stop using the cobas Liat System and contact Roche if you suspect issues


Ivermectin should not be used treat or prevent COVID-19

Ivermectin has not been approved for treating or preventing COVID-19

  •  Approved to treat people with intestinal strongyloidiasis and onchocerciasis, two conditions caused by parasitic worms
  • Different formulation used in animals to prevent heartworm disease and certain internal and external parasites 
  • FDA has not reviewed data to support use in COVID-19 patients; some initial research is underway
  • Taking ivermectin is dangerous: Interaction with other medications e.g. blood-thinners, potential to overdose with nausea, vomiting, diarrhea, hypotension (low blood pressure), allergic reactions (itching and hives), dizziness, ataxia (problems with balance), seizures, coma and even death


picture of patient and doctor

Learn about treatment options available today

Image credit: Cue, Roche, FDA

COVID-19 News: Janssen vaccine EUA, Pfizer-BioNTech vaccine flexibilities, Vaccine safety surveillance, Pulse oximeter limitations, Virus variants policies

Janssen COVID-19 Vaccine EUA

INDICATION: For the prevention of coronavirus disease 2019 for use in individuals 18 years of age and older; administered as a single dose

ADDRESSING UNMET NEED: Expands the availability of vaccines, the best medical prevention method to help us in the fight against the pandemic

MECHANISM OF ACTION: Uses modified Ad26 to deliver a piece of the DNA to make virus “spike” protein; body can temporarily make the spike protein, which does not cause disease, but triggers the immune system to learn to react defensively, producing an immune response against SARS-CoV-2



  • Ongoing randomized, placebo-controlled study, n=39,321, being conducted in South Africa, South America, Mexico, U.S, did not have evidence of SARS-CoV-2 infection prior to receiving the vaccine, Vaccine vs placebo
  • ~ 67% effective in preventing moderate to severe/critical COVID-19 occurring 14 days after vaccination
  • ~ 66% effective in preventing moderate to severe/critical COVID-19 occurring 28 days after vaccination
  • ~77% effective in preventing severe/critical COVID-19 occurring 14 days after vaccination
  • ~85% effective in preventing severe/critical COVID-19 occurring 28 days after vaccination
  • No evidence on prevention of person to person transmission


  • N= 43,783, vaccine vs. placebo, followup of 8 weeks (median)
  • Most commonly reported side effects: Pain at the injection site, headache, fatigue, muscle aches and nausea. Most of these side effects were mild to moderate in severity and lasted 1-2 days.
  • Need to report adverse safety and vaccine administration errors to iVaccine Adverse Event Reporting System (VAERS)


  • All manufacturing facilities will comply with Current Good Manufacturing Practice
  • File Certificates of Analysis (CoA) established specifications and specific results for each quality control test
  • File quarterly manufacturing reports with listing of all Drug Substance and Drug Product lots produce


  • Provide fact sheets on dosing instructions, benefits and risks providers and recipients
  • Continue clinical trials to obtain additional safety and effectiveness information and pursue approval (licensure)
  • Conduct post-authorization observational studies to evaluate adverse events of special interest, along with deaths and hospitalizations, and severe COVID-19


close up of syringe

More Flexible Storage, Transportation Conditions for Pfizer-BioNTech COVID-19 Vaccine

Undiluted frozen vials can be transported and stored at conventional freezer temperatures

  • Based on data to demonstrate stability after storage up to two weeks at standard freezer temperature
  • Will help ease burden of procuring ultra-low cold storage equipment and help get vaccine to more sites


Vaccine Safety and Surveillance

Webpage to provides overview of surveillance systems to monitor safety of authorized COVID-19 vaccines

  • Data from FDA’s CBER + CDC + CMS + VA + other academic and large non-government healthcare data systems
  • Includes international pharmacovigilance efforts, including International Coalition of Medicines Regulatory Authorities (ICMRA) and the World Health Organization (WHO)

Passive surveillance: Reviewing data from FDA’s Vaccine Adverse Event Reporting System (VAERS) , CDC’s  v-safe text-based monitoring system 

Active surveillance: Analyzing large healthcare data systems such as Sentinel BEST (Biologics Effectiveness and Safety) System and the CMS system


Pulse Oximeter Accuracy and Limitations

Pulse oximeters may be less accurate in people with dark skin pigmentation

  • Risk that an inaccurate measurement may result in unrecognized low oxygen saturation levels
  • Important to understand the limitations of pulse oximetry and how accuracy is calculated and interpreted
  • Prescription oximeters are reviewed and authorized by the FDA an used in hospitals and doctor’s offices
  • Over-the-counter (OTC) oximeters are sold directly to consumers, are not cleared by the FDA and should not be used for medical purposes.
  • Important recommendations for patients and their caregivers on how to use pulse oximeters at home
  • Recommendations for health care providers on how to use pulse oximeters for better accuracy
  • Background on pulse oximeters and the FDA’s actions to evaluate factors that may affect pulse oximeter accuracy and performance
  • Instructions for reporting problems with pulse oximeters to the FDA


Policies for Addressing Virus Variants: Vaccines, Diagnostics and Therapeutics

Guidances for medical product developers, specifically covering vaccines, diagnostics and therapeutics products

Vaccines : Updated October 2020 guidance, Emergency Use Authorization for Vaccines to Prevent COVID-19

COVID-19 Tests: New guidance for test developers, Policy for Evaluating Impact of Viral Mutations on COVID-19 Tests

Therapeutics: New guidance, Development of Monoclonal Antibody Products Targeting SARS-CoV-2, Including Addressing the Impact of Emerging Variants, During the COVID-19 Public Health Emergency. Revised guidance COVID-19: Developing Drugs and Biological Products for Treatment or Prevention.


Image credit: FDA, CDC

Marketing Authorizations: TALUS SPACER for ankle avascular necrosis, eXciteOSA for mild obstructive sleep apnea, COSELA for chemotherapy-induced myelosuppression

Patient Specific TALUS SPACER

Additive Orthopaedics, LLC.

INDICATION FOR USE: For avascular necrosis (AVN)of the ankle joint

The anatomical landmarks necessary for the design and creation of the Spacer must be present and identifiable on computed tomography scan


  • AVN is a rare condition that causes the death of bone tissue due to lack of blood supply
  • Implant provides joint-sparing alternative to other surgical interventions commonly used in late-stage AVN


  • Solid, polished replica of the patient’s bone to allow patient to regain motion and reduce pain without amputation
  • Implant is 3D printed in CoCr using a laser sintering platform
  • All surfaces of the device are hand polished to a mirror finish
  • Implant is cleaned and passivated per ASTM A967 and will be sterilized by the end user via steam sterilization.
  • Creation of a patient-specific device is achieved via computed tomography (CT) scanning and CAD/CAM generation
  • No specific instruments are provided with the subject device


  • 31 patients and 32 talus replacement surgeries (one patient had operations on both ankles) with the implant
  • Primary probable benefit endpoint: Reduction in baseline level pain following surgery using the Visual Analog Scale (“VAS”) for pain; secondary endpoints of ankle range of motion, Foot and Ankle Outcome Scores
  • Improvement in scores after surgery with further improvement 2 and 3 years after surgery
  • Few safety events : Pain and scare tissue; most significant risk is reoperation

REGULATORY PATHWAY: Humanitarian Device Exemption

  • Humanitarian Use Designation (HUD); intended to benefit patients for condition that affects not more than 8,000 individuals in the U.S. per year
  • Post-Approval Study (PAS) is a prospective post-approval US registry study to provide ongoing safety and probable benefit assessment



Signifier Medical Technologies, LLC.

INTENDED USE: For the reduction of snoring and obstructive sleep apnoea by strengthening tongue muscles via electrical muscle stimulation

INDICATIONS FOR USE: For use to reduce snoring and mild sleep apnoea (AHI<15) for patients that are 18 years or older


  • OSA has potential serious long-term effects such as heart attack, glaucoma, diabetes, cancer and cognitive and behavioral disorders
  • First device used while awake that is intended to improve tongue muscle function, which in time can help prevent the tongue from collapsing backwards and obstructing the airway during sleep


  • Removable tongue muscle stimulation device that delivers neuromuscular stimulation to the tongue
  • Through a mouthpiece that sits around the tongue; has four electrodes, two located above the tongue and two located below the tongue
  • Provides electrical muscle stimulation action in sessions that consist of a series of electrical pulses with rest periods in between
  • Used for 20 minutes once a day during a wakeful state, for a period of 6‐weeks, and once a week thereafter


  • Clinical study, n= 115 patients with snoring, including 48 patients with snoring and mild OSA
  • Endpoint: 20% time reduction in snoring at levels > 40dB in 87 patients, 48% reduction in Apnea-Hypopnea Index in 41 patients with OSA
  •  Most common adverse events: Excessive salivation, tongue or tooth discomfort, tongue tingling, dental filling sensitivity, metallic taste, gagging and tight jaw
  • Patients should receive a comprehensive dental examination prior to use of the device

REGULATORY PATHWAY: De Novo classification


COSELA (trilaciclib) injection

G1 Therapeutics

INDICATION FOR USE: To decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan containing regimen for extensive-stage small cell lung cancer (ES-SCLC)


  • Protecting bone marrow function may help make their chemotherapy for ES-SCLC safer and improve treatment compliance
  • Treatment option can reduce the occurrence of a common, harmful side effect of chemotherapy

MECHANISM OF ACTION: Transient inhibitor of CDK 4 and 6. Hematopoietic stem and progenitor cells
(HSPCs) in the bone marrow give rise to circulating neutrophils, RBCs, and platelets. HSPC proliferation is dependent on CDK4/6 activity


  • Three randomized, double-blind, placebo-controlled trials, n=245 patients, COSELA vs placebo
  • Primary endpoint: Proportion of patients with severe neutropenia and duration of severe neutropenia in the first cycle of chemotherapy
  • COSELA resulted in lower chance of severe neutropenia vs, placebo; also reduced duration of sever neutropenia
  • Most common side effects: Fatigue; low levels of calcium, potassium and phosphate; increased levels of an enzyme called aspartate aminotransferase; headache; and infection in the lungs (pneumonia)
  • Patients should also be advised about injection site reactions, acute drug hypersensitivity, interstitial lung disease/pneumonitis (lung tissue inflammation) and embryo-fetal toxicity


  • Priority Review and Breakthrough Therapy designations


Image credit: Additive Orthopaedics, Signifier Medical, G1 Therapeutics

News and Views: Novel device innovation, Alternative nonclinical testing, Technology Modernization Action Plan, CDRH regulatory science tools, Drug safety priorities, CDER ISTAND program

Novel Device Innovation Despite COVID-19 Challenges

FDA approved, cleared, or authorized (“authorized”) 132 novel medical devices in 2020 (vs. 29 novel devices authorized in 2010)

  • Novel devices include those brought to market through PMA, HDE De Novo pathways and only those 510(K) with Breakthrough designation. Also first-of-a-kind devices authorized under EUA authority
  • Devices have ‘new’ technology AND address an unmet need, or may be safer or more effective than currently available alternatives

Examples include:

  • Game-based digital therapeutic to improve attention function in children with ADHD
  • Anterior cruciate ligament (ACL) implant as an alternative to ACL reconstruction to treat ACL tears
  • Continuous renal replacement therapy device for a lower weight pediatric population
  • Liquid biopsy next-generation sequencing companion diagnostic test
  • Cardiac ultrasound software that uses artificial intelligence (AI) for quality diagnostic images 
  • Automated insulin delivery and monitoring system for use in young pediatric patients
  • High density lipoprotein plasma delipidation system to reduce coronary artery atheroma in certain adult patients
  • High intensity focused ultrasound (HIFU), magnetic-resonance guided, ablation system for osteoid osteomas
  • Several COVID-19 specific devices for diagnosing and testing


Advancing Alternative Methods for nonclinical testing

Development of new regulatory approaches that can help improve predictivity–and potentially replace, reduce and/or refine animal testing

  • Leveraging advances in systems biology, stem cells, engineered tissues, and mathematical modeling
  • Enhancing ability to predict risk and efficacy
  • Examples include Microphysiological System and Organ-on-a-chip


Technology Modernization Action Plan (TMAP) to Accelerate Regulatory Decisions

TMAP approach to the use of technology for review of medical product applications and create a nimble workforce – with 3 components:

  • Modernization of the FDA’s technical infrastructure and operations
  • Enhancing FDA’s capabilities to develop technology products
  • Communication and collaboration with external stakeholders to drive technological progress


CDRH Regulatory Science Tools to Help Assess New Medical Devices

Catalog of tools developed by CDRH Office of Science and Engineering Labs (OSEL) to help improve development and assessment of emerging medical technologies

  • Should be evaluated for suitability within specific context of use
  • Tool categories : Phantoms, Methods, Computational Models and Simulations


CDER Drug Safety Priorities

Report of CDER’s work to manage drug safety issues through modernized safety surveillance methods and innovative responses to safety concerns

  • Key safety-related milestones and accomplishments of 2020
  • Initiatives for promoting and protecting public health during the COVID-19 pandemic
  • Ongoing programs such as Adverse Event Reporting System, Sentinel System, and Safe Use Initiative
  • Ongoing activities to address the national opioid crisis, cancer-causing impurities in medicines, Drug Safety Communications tools


Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program

Use of Drug Development Tools (DDTs) -methods, materials, or measures- that have the potential to facilitate drug development

  • ISTAND Pilot Program to expand DDT types by encouraging development of DDTs that are out of scope for existing DDT qualification programs but may still be beneficial for drug development


  • Tools that may help enable remote or decentralized trials
  • ​Tools that may advance our understanding of drugs
  • Tools that leverage digital health technologies


Image credit: FDA

COVID-19 News: Janssen vaccine review, Impact of new virus variants, Bamlanivimab and Etesevimab for treatment, Convalescent Plasma EUA update

Advisory Committee Meeting to Discuss Janssen Biotech Vaccine

 Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Feb. 26, 2021

  • Discuss data submitted in support of safety and effectiveness for EUA for a COVID-19 vaccine
  • VRBPAC members are independent, scientific and public health experts who advise FDA on the safety and effectiveness data
  • Final EUA decision made by FDA


Medical Product Development to Address New Virus Variants

FDA actively assessing impact of new strains on safety and effectiveness of authorized products

  • Guided by prior experience with evolving infectious diseases e.g. Influenza, HIV
  • Communicating with medical product sponsors on impact of COVID-19 variants on their products
  • New therapeutics: Focus on expediting virus-targeting monoclonal antibodies
  • New diagnostics: Focus on  in silico monitoring to assess for mutations and impact



Eli LIlly

INDICATION: Treatment of mild to moderate COVID-19 in adults and pediatric patients (12 years of age or older weighing at least 40 kilograms [about 88 pounds]) who test positive for SARS-CoV-2 and who are at high risk for progressing to severe COVID-19

The authorized use includes treatment for those who are 65 years of age or older or who have certain chronic medical conditions

MECHANISM OF ACTION: Monoclonal antibodies that are specifically directed against the spike protein of SARS-CoV-2, designed to block the virus’ attachment and entry into human cells; the mAbs to different but overlapping sites on the spike protein of the virus


  • Placebo-controlled clinical trial of patients with COVID-19 at high risk for disease progression
  • Single iv of mAbs significantly reduced COVID-19-related hospitalization and death during 29 days of follow-up vs. compared to placebo
  • Safety and effectiveness continue to be evaluated



Convalescent Plasma EUA updated to Reflect New Data

EUA revised to limit use of high titer COVID-19 convalescent plasma

  • For the treatment of hospitalized patients with COVID-19 early in the disease course
  • For hospitalized patients who have impaired humoral immunity and cannot produce an adequate antibody response
  • Plasma with low levels of antibodies has not been shown to be helpful in COVID-19
  • Transfusion of COVID-19 convalescent plasma in hospitalized patients late in the course of illness (e.g. following respiratory failure requiring intubation and mechanical ventilation) has not been associated with clinical benefit


Image credit: FDA

COVID-19 News: Acting FDA Commissioner, Moderna vaccine and other FAQs, Vaccine distribution tool, Hand sanitizer safety and use, Authorized tests and collection kits, Fraudulent products

Janet Woodcock was named Acting Commissioner of FDA

Will oversee the full breadth of the FDA portfolio and execution of the Federal Food, Drug, and Cosmetic Act and other applicable laws

  • Safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices
  • Safety and security of food supply, cosmetics, dietary supplements, products that give off electronic radiation
  • Regulation of tobacco products



Moderna vaccine : EUA issued on Dec 18, 2020

  • Data supporting EUA
  • Vaccine effectiveness and safety
  • Results in racial, high risk ethnic groups, breastfeeding women
  • Side effects, allergic reactions, reporting of side effects and additional safety monitoring
  • Interchangeability with Pfizer-BioNTech vaccine

Other topics

  • Hand sanitizers
  • Testing
  • Medical glove 
  • Surgical mask and gown shortage
  • 3D Printing of Medical Devices & Parts
  • Ventilators
  • Manufacturing, Supply Chain, and Drug Inspections
  • Food Safety
  • Animal Food Safety
  • Face Mask and Surgical Mask



21 FORWARD for Vaccine Distribution to Food and Agriculture Workers

New data analysis tool, 21 FORWARD, to identify food supply interruptions can be used for planning vaccine distribution

  • Will augment other data sources on food and agriculture workers
  • Will provide state and local officials with information to help determine count and location of food and agriculture workers    


Hand Sanitizer Safety and Use Communication Toolkit

FDA communication materials for consumers, patients, and health care professionals to help increase hand sanitizer safety


Authorized COVID-19 Tests and Collection Kits

 To date, the FDA has authorized 309 tests and sample collection devices

  • 235 molecular tests and sample collection devices
  • 63 antibody tests
  • 11 antigen tests
  • Tests that can be used in laboratories, in doctors’ offices and at home


Fraudulent Coronavirus Disease 2019 (COVID-19) Products

Enforcement actions against firms for selling fraudulent products with claims to prevent, treat, mitigate, diagnose or cure COVID-19

  • Actively monitoring for firms marketing products with fraudulent COVID-19 prevention and treatment claims
  • To protect consumers from firms selling unapproved products and making false or misleading claims
  • Warning letters, seizures, injunctions or criminal prosecutions for violating the law


Image credit: FDA

2021 FDA Vision: Regulatory science focus areas, AI/ML action plan, Advanced manufacturing, 2020 drug approval learnings, Device regulatory flexibility

2021: Focus Areas of Regulatory Science (FARS)

Four focus areas for regulatory science research for innovative products, to inform regulatory decision-making and improve sponsor guidance to sponsors

  • Public Health Preparedness and Response: Medical countermeasures and preparedness, Reduce pathogen contamination, Substance Use disorders, Antimicrobial resistance, Food safety, Compounded drugs
  • Increasing Choice and Competition through Innovation: Individualized therapeutics and Precision Medicine, Complex innovative trial design, Microbiome research, Novel foods and food ingredients, Regenerative medicine, Advanced manufacturing, Generic alternatives for complex drugs, Product Development Tools
  • Unleashing the Power of Data: Product safety surveillance, Diverse data and technologies (AI, digital health, real-world evidence
  • Empowering Patients and Consumers: Preferences and perspectives, PROS and COAs, make better-informed decisions


Artificial Intelligence/Machine Learning (AI/ML)-Based Software as a Medical Device (SaMD) Action Plan

Total product lifecycle-based regulatory oversight to deliver safe and effective software functionality for AI/ML-based SaMD

  • Update proposed framework for AI/ML-based SaMD, including through issuance of draft guidance on the Predetermined Change Control Plan
  • Encourage harmonization of Good Machine Learning Practice (GMLP) development
  • Hold public workshop on device labeling to support transparency to users and enhances trust in AI/ML-based devices
  • Develop methodology for evaluation and improvement of ML algorithms, including identification and elimination of bias, evaluation and promotion of algorithm robustness
  • Piloting Real-World Performance (RWP) process for AI/ML-based SaMD


Advanced Manufacturing Technologies

Importance of regulatory programs for advancing manufacturing, computational modeling, and other emerging technologies

  •  New collaboration with the National Institute of Standards and Technology (NIST)  to increase U.S. medical supply chain resilience and manufacturing of drugs, biological products, medical devices through adoption of 21st century manufacturing technologies
  •  New initiatives for advanced manufacturing : CDER Emerging Technology Program, CDRH Case for Quality, CBER Advanced Technologies Program


Learnings from 2020 New Drug Therapy Approvals

Meeting challenges to respond to the COVID-19 pandemic while bringing safe and effective new drug therapies for a range of other diseases and conditions

  • Innovation across different medical conditions:  Infectious diseases, Neurological advances, Heart, blood, kidney, and endocrine disease advances, Autoimmune conditions, wide array of cancers
  • New therapies for rare diseases: Including ebola and rare cancers
  • Review efficiencies: Meeting PDUFA goals, first cycle approvals, expedited programs


Medical Device Regulatory Flexibility

Medical device enforcement policies responsive to public health emergency (PHE)

  • Review of products relevant to PHE
  • Review of MAUDE database for adverse events reports related to use during pandemic
  • Based on de minimis number of adverse events, immediately exempting 7 devices from the premarket notification requirement, and proposal to exempt an additional 84 devices after public comment is closed


Image credit: FDA

COVID-19 News: MODERNA vaccine, App Design-a-thon winners, Holiday food safety tips


ModernaTX, Inc

INDICATION: Prevention of COVID-19 in individuals 18 years of age and older

  • Administered via intramuscular injection as a series of two doses, one month apart

ADDRESSING UNMET NEED: Second vaccine to combat the pandemic

MECHANISM OF ACTION: Contains a small piece of the SARS-CoV-2 virus’s mRNA that instructs cells in the body to make the virus’s distinctive “spike” protein; triggers the immune system to learn to react defensively, producing an immune response against SARS-CoV-2  


  • Ongoing randomized, placebo-controlled U.S. study, n=28,207 participants without SARS CoV-2 infection
  • 94.1% effective in preventing COVID-19 disease; 11 cases in vaccine group vs. 185 in placebo with 30 severe cases
  • Data are not available to determine how long the vaccine will provide protection
  • No available evidence of prevention of person to person transmission
  • Most commonly reported side effects: Pain at the injection site, tiredness, headache, muscle pain, chills, joint pain, swollen lymph nodes in the same arm as the injection, nausea and vomiting, and fever; mostly after second dose


  • Pharmacovigilance plan to the FDA to monitor the safety and complete complete longer-term safety follow-up
  • Continue clinical trials to obtain additional safety and effectiveness information and pursue approval (licensure)

FDA website

Translations of Fact Sheet

CDC information

Design-a-thon for COVID-19 At-Anywhere Diagnostic Tests

AIM: To increase speed, quality, comprehensiveness, and utility of SARS-CoV-2 diagnostic test data


Oracle: COVID-19 Immutable Test Results Submission and Visualization

Interpret-COVID: Consumer Dx Test App

Net Medical Xpress Solutions, Inc: Net Medical’s Telemed for COVID-19 Wireless Data 



FDA Food Safety tips for the holidays

COVID-19 News: Moderna Vaccine update, Pfizer vaccine FAQ, BinaxNOW home test, ELLUME home test, PIXEL home sample collection

Moderna Vaccine: To receive FDA EUA

  • Positive Vaccines and Related Biological Products Advisory Committee Meeting
  • FDA has informed Moderna about finalization and issuance of EUA
  • FDA has notified CDC and and Operation Warp Speed to execute plans for timely vaccine distribution   


Pfizer-BioNTech COVID-19 Vaccine FAQs

  • New webpage to cover specifics, such as what data did the FDA use to make the decision to authorize the vaccine for emergency use, to more general questions, such as how does a vaccine go from emergency use authorization to licensure

FAQ page

 BinaxNOW COVID-19 Ag Card Home Test   


  • For prescription use at home with self-collected nasal swab samples, 15 years or older
  • Also for use with adult-collected nasal swab samples, 4 years or older
  • Antigen test that detects fragments of proteins from SARS-CoV-2
  • eMed portal and certified guide to support testing process to ensure a successful testing experience
  • Results within 20 minutes; also shared with public health authorities to monitor and manage virus spread
  • $25 / test




  • First over-the-counter (OTC) fully at-home diagnostic test for COVID-1
  • Rapid, lateral flow antigen test that detects fragments of proteins of the SARS-CoV-2 virus from a nasal swab sample
  • From any individual 2 years of age or older
  • Can be sold in drug stores, where a patient can buy it, swab their nose, run the test and find out results in 20 minutes
  • Mobile app requires individuals to input their zip code and date of birth and reports results to public health authorities to monitor disease prevalence

EUA Letter

PIXEL Home Collection Kit


  • Allows a person to self-collect a nasal sample in their home and then send to LabCorp for testing
  • Can be used by anyone aged 18 or over, and purchased online or in a store without a prescription
  • For use with LabCorp’s COVID-19 RT-PCR Test

EUA Letter

Image credits: FDA, Abbott, Ellume, LabCorp

Emergency Use Authorization for First COVID-19 Vaccine

Pfizer-BioNTech COVID-19 Vaccine


INDICATION & DOSING : For active immunization to prevent COVID-19 in individuals 16 years of age and older

  • Administered as a series of two doses, three weeks apart

DESCRIPTION: Contains SARS-CoV-2 virus’s mRNA that instructs cells to make virus’s distinctive “spike” protein; does not cause disease, but triggers the immune system to produce immune response against SARS-CoV-2    


  • Ongoing randomized, placebo-controlled international study, vaccine vs placebo
  • Analysis of 36,523 participants, majority from US, did not have evidence of SARS-CoV-2 infection through seven days after the second dose
  • 95% effective in preventing COVID-19 disease; 8 cases (1 severe) with vaccine vs 162 (3 severe) in placebo
  • Data not available to conclusively determine duration of protection or prevention of person-to-person transmission


  • Most commonly reported side effects: Pain at the injection site, tiredness, headache, muscle pain, chills, joint pain, and fever; more people experienced side effects after the second dose  
  • Mandatory FDA reporting of all vaccine administration errors, serious adverse events, cases of Multisystem Inflammatory Syndrome (MIS), and cases of COVID-19 that result in hospitalization or death

REG PATHWAY: Emergency Use Authorization

  • Pharmacovigilance plan to monitor and complete longer-term safety follow-up for enrolled patients
  • Continue clinical trials to obtain additional safety and effectiveness information and pursue approval (licensure)

COVID-19 News: Vaccine Advisory Committee for Pfizer vaccine, RC COVID-19 +Flu RT-PCR for COVID-19 + Flu test, Precision immunology App-a-thon, Warning about face masks and MRI

Vaccines and Related Biological Products Advisory Committee December 10, 2020 Meeting Announcement

The Center for Biologics Evaluation and Research’s (CBER), Vaccines and Related Biological Products Advisory Committee (VRBPAC) will meet in open session to discuss Emergency Use Authorization (EUA) of the Pfizer-BioNTech COVID-19 Vaccine for the prevention of COVID-19 in individuals 16 years of age and older.

Speaker presentations will be available by 8 AM on Dec 10, 2020

The online web conference meeting will be available at the following:

FDA Briefing Document

Pfizer Briefing Document

FDA Authorizes First COVID-19 and Flu Combination Test for use with home-collected samples


Quest diagnostics

INDICATION FOR USE: Simultaneous qualitative detection and differentiation of nucleic acid from SARS-CoV-2, influenza A virus and/or influenza B virus in nasal swab specimens self-collected at home using the Quest Diagnostics Self-Collection Kit for COVID-19 +Flu by individuals suspected of respiratory viral infection

Clinical signs and symptoms of respiratory viral infection due to SARS-CoV-2 and influenza can be similar. Specimens collected using the Quest Diagnostics Self-Collection Kit for COVID-19 +Flu can be transported at ambient temperature for testing

Under a health care provider’s order, patients can collect a sample at home and ship it to a Quest Diagnostics laboratory for analysis following the instructions included with the self-collection kit.

REG PATHWAY: Emergency Use Authorization


 COVID-19 Precision Immunology App-a-thon

To develop innovative applications to explore the relationship between personalized immune repertoires and COVID-19 disease variables and associated factors

  • Phase I: Extracting knowledge from molecular sequencing data from T Cell Receptors/B Cell Receptors and associated clinical variables. Facilitate understanding of relationship between personalized immune repertoires and COVID-19 disease characteristics such as severity and associated factors. For participants from the general data science community and the computational immunology community
  • Phase II: Create research tools and apps to report the outputs from Phase I to be incorporated into an easily interpretable format for healthcare professionals

Deadline: January 29, 2021.


Reminder patients should not wear any metal during an MRI

Patients may be injured by face masks with metal parts and coatings during a Magnetic Resonance Imaging (MRI) exam. Metal parts may become hot and burn the patient

  • Patients should not wear any metal during an MRI

If burned by face mask during an MRI, please report the event to the FDA. 

Image credit: FDA

COVID-19 News: Vaccine 101, Vaccine Advisory Committee meeting, BARICITINIB (+ Remdesivir) for treatment, LUCIRA at-home test kit, CASIRIVIMAB + IMDEVIMAB for treatment, Bioburden reduction systems, Non-invasive remote monitoring devices

Vaccine Development – 101

Center for Biologics Evaluation and Research (CBER) ensures rigorous scientific and regulatory processes and review.

Typical process
It Starts in a Lab

Research Moves Forward

Testing the Vaccine in People

Special Considerations – Public health emergencies and more information

Assessment of Manufacturing is Also a Key Component

Seeking Approval

Prescribing Information/Labeling

FDA Oversight Continues After Approval

FDA Research Provides a Unique Perspective


Dec 10, Advisory Committee Meeting to Discuss COVID-19 Vaccine Candidate

Vaccines and Related Biological Products Advisory Committee (VRBPAC) to discuss EUA of COVID-19 vaccine from Pfizer, Inc. in partnership with BioNTech Manufacturing GmbH

  • Public Meeting with outside scientific and public health experts from around the country
  •  Discussion about safety and effectiveness of vaccine
  • Help ensure clear public understanding of the scientific data and information that the FDA will evaluate in order to make a decision about whether to authorize a vaccine for emergency use for the prevention of COVID-19.”



Eli Lilly ( and Gilead)

INDICATION: Treatment of suspected or laboratory confirmed COVID-19 in hospitalized adults and pediatric patients two years of age or older requiring supplemental oxygen, invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).


MECHANISM OF ACTION: Janus kinase inhibitor (JAK) which blocks enzymes interfering with the pathway that leads to inflammation


  • Based on totality of evidence
  • Randomized, double-blind, placebo-controlled clinical trial conducted by National Institute of Allergy and Infectious Diseases (NIAID), n=1,0333 with moderate-severe COVID-19, baricitinib + remdesivir vs placebo + remdesivir, followup for 29 days
  • Primary Endpoint: Recovery defined as either being discharged from hospital or being hospitalized but not requiring supplemental oxygen and no longer requiring ongoing medical care; 7 days for baricitinib + remdesivir vs. 8 days for placebo + remdesivir.
  • Odds of patient’s condition progressing to death or being ventilated at day 29 was lower in the baricitinib + remdesivir group vs placebo + remdesivir group (statistically significant)
  • Similarly, odds of clinical improvement at day 15 was higher in the baricitinib + remdesivir group (statistically significant)

REGULATORY PATHWAY: Emergency Use Authorization

  • Previously approved for treatment of moderately to severely active rheumatoid arthritis
  • Expedited by Coronavirus Treatment Acceleration Program (CTAP)

Fact Sheet

LUCIRA COVID-19 All-In-One Test Kit 

Lucira Health

INDICATION FOR USE: Single-use test kit intended to detect SARS-CoV-2 that causes COVID-19. This test is authorized for prescription home use with self-collected nasal swab samples in individuals aged 14 and older who are suspected of COVID-19 by their healthcare provider.

Test is authorized for use at the Point of Care (POC), in patient care settings operating under a CLIA Certificate of Waiver, Certificate of Compliance, or Certificate of Accreditation

Test utilizes a molecular amplification technology for the detection of SARS-CoV-2 RNA in individuals with known or suspected COVID-19. Positive results are indicative of the presence of SARS-CoV-2. Individuals who test positive should self-isolate and seek additional care from their healthcare provider.

Negative results do not preclude SARS-CoV-2 infection. Individuals who test negative and continue to experience COVID-like symptoms should seek follow up care from their healthcare provider.


  • Analytical sensitivity based on Limit of detection, Inclusivity, Cross-reactivity,
  • Human usability study, n=398 asymptomatic subjects, for safe and effective use
  • Community testing study, n=101 asymtomatic subjects, independent collection of Lucira nasal swab samples and running the test. Comparable results vs high sensitivity molecular FDA Authorized SARS-CoV-2 assay (Roche Cobas)

ADDRESSING UNMET NEED: First COVID-19 diagnostic test for self-testing at home and that provides rapid results

REG PATHWAY: Emergency Use Authorization

Instructions for Use

CASIRIVIMAB + IMDEVIMAB intravenous infusion 


INDICATION: Treatment of mild to moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progressing to severe COVID-19 and/or hospitalization. This includes those who are 65 years of age or older or who have certain chronic medical conditions.

ADDRESSING UNMET NEED: Pandemic treatment option that may help outpatients avoid hospitalization and alleviate the burden on health care system

MECHANISM OF ACTION: Monoclonal antibodies that are specifically directed against the spike protein of SARS-CoV-2, designed to block the virus’ attachment and entry into human cells


  • Randomized, double-blind, placebo-controlled clinical trial, n= 799 non-hospitalized adults with mild to moderate COVID-19 symptoms
  • Primary endpoint: Time-weighted average change in viral load from baseline; viral load reduction in patients treated with casirivimab and imdevimab was larger than placebo
  • Secondary endpoint: Hospitalizations and emergency room visits within 28 days after treatment; 3% of casirivimab and imdevimab-treated patients vs. 9% on placebo
  • Possible side effects: Anaphylaxis and infusion-related reactions, fever, chills, hives, itching and flushing. 

REGULATORY PATHWAY: Emergency Use Authorization

  • Part of Coronavirus Treatment Acceleration Program


Bioburden Reduction Systems

Bioburden: Whole population of viable microorganisms that inhabit a non-sterile surface or device

 Reduction : Dry heat, such as application of temperatures of 70° C for 60 minutes or 75° C for 30 minutes, will kill many of the moderately-resistant microbes present

Intention: To kill and reduce microorganisms on a non-sterile device surface. For use in addition to existing CDC reuse recommendations

Use: In shortage situations, when there is an insufficient supply of essential supplies such as new respirators


Non-Invasive Remote Monitoring Devices Used to Support Patient Monitoring During the pandemic

Policy to expand availability and capability of non-invasive remote monitoring devices

  • To facilitate patient monitoring while reducing patient and healthcare provider contact
  • To remain in effect only for the duration of the COVID-19 public health emergency
  • Enforcement policy applies to legally marketed non-invasive remote monitoring devices that measure or detect common physiological parameters to support patient monitoring
  • Devices can be connected to wireless network to transmit patient’s measurements directly to health care provider


Image credit: FDA, Lucira Health, Regeneron

COVID-19 News: BAMLANIVIMAB monoclonal antibody therapy, cPass neutralization antibody detection kit, Enhancing clinical trial diversity, Policy for diagnostic tests, Coronavirus Treatment Acceleration Program, Fraudulent flu products

bamlanivimab investigational monoclonal antibody therapy

Eli Lilly

INDICATION: For patients with positive results of direct SARS-CoV-2 viral testing who are 12 years of age and older weighing at least 40 kilograms, and who are at high risk for progressing to severe COVID-19 and/or hospitalization

ADDRESSING UNMET NEED: Monoclonal antibody for coronavirus treatment

MECHANISM OF ACTION: Monoclonal antibody that is specifically directed against the spike protein of SARS-CoV-2, designed to block the virus’ attachment and entry into human cells


  • Interim analysis from Phase II randomized, double-blind, placebo-controlled clinical trial, n=465 non-hospitalized adults with mild to moderate COVID-19 symptoms; several doses of bamlanivimab vs placebo
  • Primary endpoint: Change in viral load from baseline to day 11; similar outcomes
  • Secondary endpoint: COVID-19-related hospitalizations or emergency room visits within 28 days after treatment; 3% for bamlanivimab vs.10% for placebo

REGULATORY PATHWAY; EUA, Coronavirus Treatment Acceleration Program

Fact Sheet

cPass SARS-CoV-2 Neutralization Antibody Detection Kit


INDICATION: Qualitative detection of total neutralizing antibodies to SARSCoV-2 in human serum and K2-EDTA plasma. Intended for use as an aid in identifying individuals with an adaptive immune response to SARS-CoV-2, indicating recent or prior infection

Emergency use of this test is limited to authorized laboratories

ADDRESSING UNMET NEED: First serology test authorized to detect neutralizing antibodies from recent or prior COVID-19 infection

ASSAY PRINCIPLE: Blocking Enzyme-Linked Immunosorbent Assay (ELISA) intended for qualitative direct detection of total neutralizing antibodies to SARS-CoV-2 in human serum and K2-EDTA plasma

Instruction For Use

FDA Guidance: Enhancing the Diversity of Clinical Trial Populations

Clinical trials design of drugs and biologics to include different demographic characteristics (e.g., sex, race, ethnicity, age) and non-demographic characteristics (e.g., patients with organ dysfunction, comorbid conditions, and disabilities)

  • Addresses health disparities
  • Critical during the COVID-19 public health emergency as certain segments of the population are being affected by the virus in different ways
  • Developers of CVOID-19 treatments or vaccines for COVID-19 should include diverse populations to understand their risks or benefits across all groups


FDA Guidance: Policy for Coronavirus Disease-2019 Tests During the Public Health Emergency (Revised)

To help rapidly expand testing capacity by facilitating development and use of SARS-CoV-2 tests during the public health emergency

  • Two policies for accelerating the development of certain laboratory developed diagnostic tests for COVID-19
  • EUA submission to FDA
  • No EUA submission when test is developed under State authorities and the State takes responsibility


Coronavirus Treatment Acceleration Program (CTAP) update

Special emergency program for possible coronavirus therapies

  • Accelerate patient availability of new treatments while maintaining statutory standrads for safety and effectiveness
  • Accelerate development and publication of guidances and other information for industry on developing COVID-19-related treatments


Fraudulent Flu Products

Unproven products that claim to prevent, mitigate, treat, or cure the flu

  • Not been FDA approved for safety and effectiveness; can be ineffective and dangerous
  • Might cause people to delay, forgo, or stop the medical treatment they need, leading to serious and life-threatening harm
  • Ingredients could lead to unexpected side effects and interactions with other medications
  • Can be found online, including popular marketplaces, and in retail stores
  • Labeled as dietary supplements, foods, hand sanitizers, nasal sprays, or devices

READ Warning Letters page Fraudulent product views

Image credit: FDA, GenScript

Market Authorizations: NIGHWARE App for PTSD sleep disturbance, INMAZEB for Ebola treatment, REACTIV8 Implantable Neurostimulation System for pain, MAKENA withdrawal proposal

NIGHTWARE Digital Therapeutic

Nghtware Inc

INDICATION FOR USE:  Temporary reduction of sleep disturbance related to nightmares in adults 22 years or older who suffer from nightmare disorder or have nightmares from post-traumatic stress disorder (PTSD)

The device should be used in conjunction with prescribed medications for PTSD and other recommended therapies for PTSD-associated nightmares and nightmare disorder

ADDRESSING UNMET NEED: New, low-risk treatment option that uses digital technology to provide temporary relief from sleep disturbance related to nightmares in PTSD patients

DESCRIPTION: Digital therapeutic software using Apple Watch and Apple iPhone

  • Throughout the night, Apple Watch sensors monitor body movement and heart rate during sleep
  • Data are sent to the Nightware server and, using a proprietary algorithm, the device creates a unique sleep profile for the patient
  • When Nightware detects patient experiencing a nightmare based on analysis of heart rate and body movement, the device provides vibrations through the Apple Watch


  • Randomized, sham-controlled trial of 70 patients for 30 days
  • Patients in the sham group wore the device, but no vibratory stimulation was provided
  • Effectiveness was assessed by Sleep based on two versions of the Pittsburgh Sleep Quality Index scale, including a version intended for PTSD patients
  • Safety was assessed using validated measurements of suicidality and sleepiness
  • Both the sham and active groups showed improvement on the sleep scales, with the active group showing greater improvement than sham
  • Nightware is intended to be used under the supervision of a healthcare provider. Patients who have been known to “act out” during their nightmares (sleepwalking, violence) should not use Nightware

REGULATORY PATHWAY: De Novo pathway with Breakthrough Designation

INMAZEB™ (atoltivimab, maftivimab, and odesivimab-ebgn) injection

Regeneron Pharmaceuticals

INDICATION: Treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including neonates born to a mother who is RT-PCR positive for Zaire ebolavirus infection

ADDRESSING UNMET NEED: First FDA-approved treatment for Zaire ebolavirus infection in adult and pediatric patients.

MECHANISM OF ACTION:  Targets the glycoprotein on the surface of Ebola virus and blocks attachment and entry of the virus.


  • Clinical trial led by the U.S. National Institutes of Health and the Democratic Republic of the Congo (DRC) Institut National de Recherche Biomédicale
  • Multi-center, open-label, randomized controlled trial, n= 382 adult and pediatric patients with confirmed Zaire ebolavirus infection during an Ebola virus outbreak in 2018-2019 in DRC, Inmazeb (50 mg of each monoclonal antibody) vs control
  • Primary efficacy endpoint: 28-day mortality
  • With Inmazeb, 33.8% died after 28 days vs. 51% on control


  • Most common: fever, chills, tachycardia (fast heart rate), tachypnea (fast breathing), and vomiting; however, these are also common symptoms of Ebola
  • Avoid concurrent administration of a live vaccine due to the treatment’s potential to reduce the vaccine’s efficacy.


  • Orphan Drug designation,  Breakthrough Therapy designation 


REACTIV8 Implantable Neurostimulation System 

Mainstay Medical

INDICATON FOR USE: For bilateral stimulation of the L2 medial branch of the dorsal ramus as it crosses the L3 transverse process as an aid in the management of intractable chronic low back pain associated with multifidus muscle dysfunction, as evidenced by imaging or physiological testing in adults who have failed therapy including pain medications and physical therapy and are not candidates for spine surgery.

ADDRESSING UNMET NEED: Treatment option for patients who have failed therapy including pain medications and physical therapy and are not candidates for spine surgery 

DEVICE DESCRIPTION: The system consists of an Implantable Pulse Generator (IPG), Leads, surgical tools and accessories, Application Software, Programmer Wand, Activator, Magnet and Tunneler. The ReActiv8 IPG, Torque Wrench and Leads, and the Mainstay Tunneler have been sterilized using ethylene oxide (EO) gas.


  • International, multi-center, prospective, randomized, blinded trial; ReActiv8 System (Treatment group) vs. active Sham Control, n= 204
  • Although the primary efficacy endpoint was not met at the 120-day visit, totality of evidence supports clinical benefit
  • Additional potential secondary benefits were observed, with improvement in patient pain symptoms, as seen in the percent pain relief, and improvement in functionality measured by the Oswestry Disability Index
  • System was determined to have safe electrical and mechanical characteristics
  • Reasonable expectation of biocompatibility
  • Adverse events that were reported were consistent with those reported with the marketed spinal systems


  • Device Generic Name: Stimulator, neuromuscular, lower back muscles, totally implanted for pain relief
  • Device Procode: QLK


MAKENA (hydroxyprogesterone caproate injection) proposed withdrawal

FDA has issued a notice of opportunity for a hearing (NOOH) for withdrawal of MAKENA, marketed by Amag Pharmaceuticals  

  • Accelerated approval granted to Amag Pharmaceuticals in 2011 to reduce the risk of preterm birth in women who previously had a spontaneous (unexplained) preterm birth
  • Company required to conduct a clinical trial to confirm the drug provided clinical benefit to newborns
  • However, confirmatory study failed to substantiate efficacy
  • Health Care Professionals discuss Makena’s benefits, risks and uncertainties with their patients to decide whether to use while a final decision is being made


Image credits: Nightware, Regeneron, Mainstay, Amag

COVID-19 News: VEKLURY for COVID treatment, Advisory committee for vaccines, Evidence Accelerator for real world data, New NextGen portal for manufacturing capacity, New Sentinel website for pandemic activities

VEKLURY (remdesivir) injection


INDICATION: For adults and pediatric patients (12 years of age and older and weighing at least 40 kg) for the treatment of coronavirus disease 2019 (COVID-19) requiring hospitalization. VEKLURY should only be administered in a hospital or in a healthcare setting capable of providing acute care comparable to inpatient hospital care

ADDRESSING UNMET NEED: First treatment for COVID-19

MECHANISM OF ACTION: Inhibitor of the SARS-CoV-2 RNA-dependent RNA polymerase (RdRp), which is essential for viral replication

EFFICACY: Three randomized, controlled clinical trials that included patients hospitalized with mild-to-severe COVID-19. Recovery was defined as either being discharged from the hospital or being hospitalized but not requiring supplemental oxygen and no longer requiring ongoing medical care

  • Median time to recovery was significantly lower with Veklury vs. placebo
  • Odds of clinical improvement were also statistically significantly higher or numerically better in the Veklury group when compared to the placebo group. 

SAFETY: Possible side effects include: increased levels of liver enzymes, which may be a sign of liver injury; and allergic reactions, which may include changes in blood pressure and heart rate, low blood oxygen level, fever, shortness of breath, wheezing, swelling (e.g., lips, around eyes, under the skin), rash, nausea, sweating or shivering


  • Prior EUA approval
  • Fast Track and Priority Review designations
  • Material Threat Medical Countermeasure Priority Review Voucher
  • Postmarketing pediatric study requirement (birth to <18 years, including neonates)
  • Postmarketing study in or remdesivir resistant SARS-CoV-2 variants  

LABEL , FDA Review Summary

COVID-19 Vaccines and FDA Advisory Committee

FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) met on 10/22 to discuss development, authorization and/or licensure of COVID-19 vaccines

  • Committee members are external scientific and public health experts from around the country, specializing in fields such as immunology, virology, infectious diseases, pediatrics, vaccine development, and vaccine safety.
  • Presentations from the experts as well as FDA scientists on COVID-19 disease and vaccine development
  • Thorough discussion on studies needed to support authorization or approval, post-marketing safety studies, ongoing safety monitoring following issuance of an emergency use authorization
  • Members of the public speak and provide input
  • Overall, to help ensure clear public understanding of the data needed for FDA authorization or approval of COVID-19 vaccines, as well as plans for their continued evaluation following authorization or approval  

Read , Event materials , To view the webcast replay, visit FDA’s YouTube Channel.

COVID-19 Evidence Accelerator: An initiative launched by the Reagan-Udall Foundation for the FDA

To gather Real world data (RWD) to help advance our nation’s ability to respond to the pandemic

  • Informing and safely speeding diagnostic and therapeutic processes
  • Provide a unique venue for major data organizations, government and academic researchers, and health systems to gather and design quick-turn-around queries and share their results
  • Bring together leading experts in health data aggregation and analytics to share insights, compare results, and answer key questions about COVID-19 treatment and response as quickly as possible


CDER NextGen portal includes manufacturing capacity data for COVID-19 drugs

CDER is gathering more information about manufacturing capacity for investigational COVID-19 products

  • Portal captures information about the specific IND, manufacturer, production rates, treatment courses, and allocations 
  • To analyze and support manufacturing efforts for potential vaccines and therapeutics such that there is a capability for mass production to meet the population need


New Sentinel website for the pandemic

Launch of newly redesigned Sentinel Initiative website to increase organization and usability and assist the Sentinel community

  • Coronavirus (COVID-19) activities, to keep up with how FDA is using Sentinel activities to protect and promote public health during the COVID-19 pandemic
  • Completed ARIA assessments & impact, for a list of safety-related outcomes, results, and regulatory impacts
  • Drug assessments, where you can search materials based on medical product, safety outcome, and study type
  • Publications & presentations, so you can read up on Sentinel activities in journals and conference materials


Image credit: FDA

News and Views: Orphan products grants, Cybersecurity, Opioid disposal toolkit, Opioid Use Disorder guidance, Breast implants patient labeling, Flu vaccine

Funding to advance medical products for the treatment of rare diseases

 Orphan Drugs are for rare diseases/disorders that affect fewer than 200,000 people in the US

Six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years 

  • Stargardt disease
  • Prevention of graft versus host disease
  • Cystic fibrosis patients colonized with nontuberculosis mycobacterium
  • CD4 positive T cell neoplasms
  • Pancreatic cancer
  • Peripheral T-cell lymphoma


FDA and Cybersecurity

Medical devices are increasingly connected to the Internet, hospital networks, and other medical devices to improve health care

  • Increase the risk of potential cybersecurity threats and impact device safety and effectiveness
  • Reducing cybersecurity risks is important but challenging

Fact Sheet on FDA’s role in medical device cybersecurity

  • FDA works closely with several federal government agencies including the U.S. Department of Homeland Security
  • Manufacturers must address pre-market and post-market cybersecurity risks; make updates to strengthen cybersecurity
  • Manufacturer is responsible for software validation to address cybersecurity vulnerabilities, conduct pre-market testing including the use of OTS software

Fact sheet

Safe Opioid Disposal ‘Remove the Risk’ Outreach Toolkit

Remove the Risk raises awareness of the serious dangers of keeping unused opioid pain medicines in the home and provides information about safe disposal of these medicines

  • Free toolkit materials—public service announcements (PSAs), social media images and posts, fact sheets
  • Designed to help groups speak with others about safe opioid disposal

Remove the Risk

Guidance to Encourage Development of Novel Medicines to Treat Opioid Use Disorder (OUD)

Guidance addresses acceptable clinical endpoints for demonstrating effectiveness to treat OUD and to predict a sustained clinical benefit

  • Reductions in adverse outcomes related to OUD (mortality, emergency medical interventions, Hep C infections)
  • Change in disease status using OUD diagnostic criteria
  • Change in drug use pattern
  • Patient Reported Outcomes (e.g., improvement in sleep or mood)
  • Other outcome measures (e.g. ability to resume work, school, or other productive activity)


Breast Implants Labeling Recommendations to Improve Patient Communication

FDA guidances to help ensure that a patient receives and understands the benefits and risks of breast implants

  • Labeling for saline/silicone breast implants for breast augmentation or breast reconstruction
  • Important for patients to have benefit and risk information for a balanced discussion with their physicians
  • Includes: Boxed Warning, Patient Decision checklist, Rupture Screening recommendation, Material/Device description, Patient Device card


FDA approved Flu vaccines provide important benefits such as preventing illnesses, hospitalizations and deaths

  • Likely that flu viruses and COVID-19 will circulate together this fall and winter; flu shot will not prevent COVID-19 but help to keep you healthy and preserve health care resources for COVID-19
  • Annual vaccination to prevent flu is the best way to reduce the risk of getting the flu and spreading it to others
  • Annual shots are needed because flu viruses can change from year to year, so the vaccine is updated; FDA, WHO, CDC, and public health experts collaborate to identify likely strains
  • Typically, children and older people are most at risk of getting sick with influenza
  • FDA ensures flu vaccine is safe, effective, and of high quality


Image credits: FDA, DHS

COVID-19: CDRH response to date, Patient outreach toolkit, Laboratory safety, Vaccine EUA guidance, Medical countermeasures R&D

Center for Devices and Radiological Health’s (CDRH) Response to Coronavirus (COVID-19)

Timeline: January 1 – September 18, 2020

  • Regulatory Flexibility: EUA templates, guidance documents, policies
  • Emergency Use Authorizations: 10x medical devices the number authorized pre-2020, tests
  • Shortage Mitigation Activities: Outreach to 1,000+ manufacturing sites across 12 countries
  • Public Health Service Corps Deployment: 58 CDRH Commissioned Corps officers
  • Engagement with Stakeholders: Emails, phine queries, webinars, townhalls

Patient Outreach Toolkit

Useful COVID-19 resources for healthcare professionals to share with patients

Fraudulent Products Factsheet
(English / Español – PDF, 1.2MB)

FDA Response to COVID-19: What You Need to Know Infographic
(English / Español – PDF, 4.4MB)

Approved COVID-19 Tweets
(English / Español – PDF, 1.6MB)

A Culture of Responsibility: Laboratory Safety at the FDA

In observance on NIH’s Biosafety & Biosecurity Month, FDA is implementing the folloiwng in its labs

  • Proper use of PPE (masks, face shields, gloves, gowns, shoe covers)
  • Engineering controls (biosafety cabinets, secure rooms)
  • Administrative controls (risk assessment, pre-work approval, medical surveillance, respiratory protection programs)
  • Best practices for decontaminating surfaces and proper procedures for packing and transporting any hazardous biomaterials, including SARS-CoV-2


Industry Guidance: Emergency Use Authorization for
Vaccines to Prevent COVID-19

Data and information needed for Emergency Use Authorization (EUA) for an investigational COVID-19 vaccine that could be potentially administered to millions of individuals

  • Chemistry, manufacturing, and controls information (CMC); nonclinical data and information; and clinical data and information, as well as administrative and regulatory information

Safety and Effectiveness Information for evaluation of benefit-risk profile

  • Bioassays for assessment of clinical endpoints
  • Supportive nonclinical studies e.g., characterization of markers associated with enhanced disease, biodistribution, shedding, and attenuation)
  • Clinical
    a. Prespecified success criteria for primary efficacy endpoint (previous guidance), placebo controlled efficacy trial of at least 50%
    b. All safety with focus on serious adverse events, adverse events of special interest, and cases of severe COVID-19 among study subject
    c. Data from Phase 3 studies should include a median follow-up duration of at least two months after completion of the full vaccination regimen
  • In addition
    a. Local and systemic solicited adverse reactions for reactogenicity
    b. All safety data from database lack, n> 3,000
    c. Sufficient cases of severe COVID-19 among study subjects
    d. Sufficient cases of individuals with prior SARS-CoV-2 infection


Cellular signaling and immune correlates for SARS-CoV-2 infection

Research award to Stanford University to help inform the development and FDA review of COVID-19 medical countermeasures

  • Explain host factors contributing to coronavirus immune responses to more rapidly predict patient outcomes
  • Innovative in-depth analysis of existing tissue samples from previous clinical and nonclinical studies
  • Profile circulating immune signatures of coronavirus infection, pathology, tissue imaging, tissue viral reservoirs, how different systems in the body are affected


Image credit: FDA, Stanford University

News & Views : New drug importation pathway, Digital Health center of excellence, ‘Intended Use’ clarification, Illegal opioid websites, Youth E-cig use trend, Collaborative communities, Promoting patient safety, Cannabis Drug Master Files

Importation of Certain FDA Approved Human Prescription Drugs, Including Biological Products, and Combination Products

Potential pathway for manufacturers to commercialize an FDA-approved drug originally intended to be marketed in a foreign country and authorized for sale in that foreign country

  • The final rule allows FDA-authorized programs to import certain prescription drugs from Canada
  • Under specific conditions to ensure no additional risk to the public’s health and safety
  • Could achieve significant reduction in the cost of covered products to the American consumer
  • Guidance provides procedures to interested manufacturers


New Digital Health Center of Excellence

Digital Health Center of Excellence for the advancement of digital health technology

  • Mobile health devices, Software as a Medical Device (SaMD), Wearables medical products
  • Centralized expertise and resource for digital health technologies and policy for digital health innovators, public, FDA staff
  • Goal: Advance health care by fostering responsible and high-quality digital health innovation
  • Objectives: Connect and build partnerships, Share knowledge,  Innovate regulatory approaches 
  • Bakul Patel appointed Director


Types of Evidence Relevant to Determining the “Intended Use” of FDA-Regulated Products

To clarify the regulatory language describing the types of evidence relevant to determining a product’s intended uses

  • Any relevant source of evidence may be considered
  • However, an unapproved use, standing alone, is not sufficient
  • Examples provided in Final Rule


FDA Warns Website Operators Illegally Selling Opioids to Consumers

Warning letters to 17 website operators for illegally selling unapproved and misbranded opioids online in violation of the Federal Food, Drug, and Cosmetic Act

  • Sale without prescription
  • Lack adequate directions for use
  • Put consumers at risk of addiction, abuse and misuse
  • Can lead to overdose and death

Warning letters issued to:,,,,,,,,,,,,,, Thomas Meds,,


Encouraging Decline in Overall Youth E-Cigarette Use, However, concerning Uptick in Use of Disposable Products

New data from the 2020 National Youth Tobacco Survey (NYTS)

  • 1.8 million fewer U.S. youth currently using e-cigarettes compared to 2019
  • However, 3.6 million U.S. youth still use e-cigarettes – a public health crisis
  • Alarming uptick in use of disposable e-cigarettes (e.g. ENDS) by youth – 26.5% of high school and 15.2% of middle school students  
  • All new and existing tobacco products need to submit premarket application for FDA review for marketing authorization – DEADLINE 9/9
  • FDA will take action if products targeted to kids; warnings letters already already issued to some


Collaborative Communities: FDA Participates in 3 New Communities

Collaborative community is a private- and public-sector collaboration which can include the FDA

  • CDRH works together on medical device challenges to achieve common objectives and outcomes
  • Challenges are ill-defined or there is no consensus on the definition of the challenges
  • Challenges and outcomes are complex
  • Partners are interrelated
  • Incremental or unilateral efforts to address the challenge have been ineffective
  • Partners seek to optimize efforts, including preventing duplication of efforts
  • Better outcomes could be achieved with integrating different perspectives, experiences, resources, and expertise

Currently participates as a member of the five collaborative communities controlled by external stakeholders

  • Standardizing Laboratory Practices in Pharmacogenomics Initiative (STRIPE) 
  • International Liquid Biopsy Standardization Alliance (ILSA) 
  • Xavier Artificial Intelligence (AI) World Consortium


Efforts to Promote Patient Safety

Institute for Healthcare Improvement (IHI) and the Agency for Healthcare Research and Quality issued “Safer Together: A National Action Plan to Advance Patient Safety,” developed by the National Steering Committee (NSC) for Patient Safety. FDA actively involved with NSC.

4 approaches creating a culture of total systems safety

  • Culture, Leadership, and Governance: Commitments to safety as a core value
  • Patient and Family Engagement: Ensure meaningful partnership
  • Workforce Safety: Eliminate harm to both patients and the workforce
  • Learning System: Networked and continuous learning


Use of Drug Master Files to Further Support Cannabis Research  

Potential benefits of using Drug Master Files (DMFs) research on drugs containing cannabis and cannabis-derived compounds

  • Used to provide confidential, detailed information about facilities, processes, or articles used in the manufacturing, processing, packaging, and storing
  • Allow 3rd parties to reference material (e.g., CMC) without disclosing DMF contents to those parties
  • Permits review of DMF information by FDA to support applications submitted by one or more applicants
  • DMF holder must provide a Letter of Authorization (LOA) and can have private agreements about information sharing, but FDA will not reveal any DMF contents to the applicant.


Image credits: FDA

COVID-19: Fraudulent products, Reference panel comparative data for assays, First Point-of-Care (POC) antibody test, Senate testimony, Infusion pump umbrella EUA revoked

Video: Beware of Fraudulent Coronavirus Tests, Vaccines and Treatments

Video explains:

  • There are currently no FDA-approved drugs or vaccines to treat or prevent COVID-19
  • Products that fraudulently claim to cure, treat, diagnose, or prevent COVID-19 haven’t been evaluated by the FDA for safety and effectiveness
  • They might be dangerous to you and your family
  • Fraudulent products can be reported to the FDA



SARS-CoV-2 Reference Panel Comparative Data

From February through the middle of May, the FDA issued a total of 59 EUAs for IVDs for the qualitative detection of nucleic acid from SARS-CoV-2

  • Reference Panel established to more precisely compare the performance of assays
  • Composed of standardized material, suitable for the determination and direct comparison of analytical sensitivity and cross-reactivity of nucleic acid-based SARS-CoV-2 assays


First Point-of-Care (POC) Antibody Test for COVID-19: Assure COVID-19 IgG/IgM Rapid Test Device

EUA USE: For POC use using fingerstick blood samples

ADDRESSING UNMET NEED: Fingerstick blood samples can now be tested in POC settings like doctor’s offices, hospitals, urgent care centers and emergency rooms rather than having to be sent to a central lab for testing


  • Lateral flow assay and is authorized for use with venous whole blood, serum, plasma and fingerstick whole blood
  • The test is authorized for the qualitative detection and differentiation of antibodies against SARS-CoV-2 indicating recent or prior SARS-CoV-2 (COVID-19) infection
  • This EUA authorizes the test for direct use with fingerstick blood samples in patient care settings, like doctors’ offices, hospitals, urgent care centers, and emergency rooms, rather than the samples being sent to a central laboratory for testing
  • Serology test results should not be interpreted to mean that a patient is immune to the virus or as an indication to stop taking steps to protect themselves and others against the spread of COVID-19


Testimony: Senate Committee on Health, Education, Labor and Pensions

Hearing entitled, “COVID-19: An Update on the Federal Response.” FDA Commissioner testimony described Agency’s efforts to protect the American public, help ensure the safety, efficacy, and quality of FDA-regulated medical products, and provide industries with tools and flexibility to do the same

  • Diagnostic testing
  • Vaccine development
  • Therapeutic development
  • Medical product supply (drugs & biological products, medical devices,
  • Inspections
  • Food supply
  • Fraudulent products


Umbrella Emergency Use Authorization of Infusion Pumps  Infusion Pump Accessories Revoked

FDA issued an umbrella EUA for infusion pumps and infusion pump accessories in May

  • For use by healthcare providers to treat COVID-19 conditions caused
  • For controlled infusion of medications, total parenteral nutrition (TPN), and/or other fluids
  • Included infusion pumps with remote monitoring or remote manual control features or administration sets and other accessories
  • However, no device has been added to the list of authorized devices

Current circumstances support revocation of the umbrella EUA

  • Individual EUAs will allow for tailored indications and scopes of authorization

EUA was revoked 

Image credit: FDA

COVID-19 News: Scientific, regulatory oversight of vaccine, Guidance on vaccine development, Guidance on manufacturing, Food-Cosmetics information center, Multilingual resources

FDA’s Scientific and Regulatory Oversight of Vaccines is Vital to Public Health

Committed to making decisions guided by science and data regarding the authorization or approval of COVID-19 vaccines. Guidance on Development and Licensure of Vaccines to Prevent COVID-19 (see below), additional guidance on EUA

Importance of diversity in clinical trial participants. FDA strongly encourages enrollment of all people – including racial and ethnic minorities, older adults, pregnant women and women of childbearing age and, as appropriate, children

FDA’s career scientists and physicians facilitating development and evaluation of safe and effective vaccines. Expertise in clinical trial design and analysis, adequacy of manufacturing and facilities for producing high-quality vaccines, and post-marketing safety surveillance

Importance of being as transparent as possible.  Public meeting of Vaccines and Related Biological Products Advisory Committee on October 22


GUIDANCE: Development and Licensure of Vaccines to Prevent COVID-19

Overview of key considerations to satisfy regulatory requirements per IND regulations and licensing regulations for chemistry, manufacturing, and controls (CMC), nonclinical and clinical data through development and licensure, and for post-licensure safety evaluation

CMC : General Considerations, Manufacture, Facilities, Inspections

Nonclinical data: Toxicity Studies, Immune Response characterization, Potential for Vaccine-associated Enhanced Respiratory Disease

Clinical trials: Populations, Design, Efficacy Considerations, Statistical Considerations, Safety Considerations

Either laboratory-confirmed COVID-19 or laboratory-confirmed SARS-CoV-2 infection is an acceptable primary endpoint

Primary efficacy endpoint point estimate for a placebo-controlled efficacy trial should be at least 50%

Post-licensure safety evaluation: Pharmacovigilance, Reqd post-marketing safety studies

Diagnostic and serological assays

Additional considerations: Vaccine effectiveness, EUA


GUIDANCE: Resuming Normal Drug and Biologics Manufacturing Operations During the COVID-19 Public Health Emergency

Guidance for manufacturers as they transition from operations impacted by the public health emergency to normal manufacturing operations. Recommendations include:

  • Addressing deviations from established cGMP activities
  • Risk Management and other important elements of a plan to resume normal drug manufacturing
  • Prioritizing activities to resume normal drug manufacturing


Food and Cosmetics Information Center (FCIC) Answers Your Questions

FCIC provides information and answers questions related to nutrition, safety, labeling of food, dietary supplements and cosmetics. Pandemic related, Complaints, Safety and Labeling, Expert advice


Multilingual COVID-19 Resources

CDC COVID-19 Communication Toolkit: For Migrants, Refugees, and Other Limited-English-Proficient Populations in various languages

Image credit: FDA

Other News and Views: Detection of nitrosamine contaminants, Trial stimulation before spinal cord stimulator implantation, Tanning beds and booths, Right to Try act, Naloxone prescribing

Rigorous Detection of Nitrosamine Contaminants in Metformin Products: Balancing Product Safety and Product Accessibility

Since 2018, multiple drug products, including angiotensin receptor blockers (ARBs), histamine blocker ranitidine (commonly known as Zantac), have been recalled due to the presence of nitrosamines at unacceptable amounts

  • CDER scientists have developed and publicly shared gas chromatography-mass spectrometry (GC-MS) and liquid chromatography-MS (LC-MS) technologies
  • For detecting and quantifying up to eight different nitrosamines  at levels below acceptable U.S. intake limit for the nitrosaminat low levels


Conduct a Trial Stimulation Period Before Implanting a Spinal Cord Stimulator (SCS) 

Implanted SCS are an aid to cope with unmanageable, chronic chronic pain

  • However, FDA continues to receive reports of associated serious side effects

Health care providers should conduct a trial stimulation period with patients to confirm satisfactory pain relief before implanting a spinal cord stimulator (SCS)

  • Implant only in patients who have passed stimulation trial performed for 3-7 days showing 50% percent reduction in pain symptoms.
  • Discuss the benefits and risks of the different types of implants and other treatment options with patients
  • Several other recommendations


Tanning Beds and Booths

Tanning beds and booths are sunlamp products used for indoor UV skin tanning

  • Risk of fire due to improper maintenance, dirty air filters blocking air flow, incompatible parts, failure to perform servicing and maintenance
  • Owners and operators should perform maintenance recommended by product manufacturers to reduce risk of smoke and fire
  • FDA monitoring adverse event reports and will keep public informed


New Rule on Reporting Requirements for Right to Try Act

Right to Try Act provides pathway for patients with life-threatening diseases who have tried all approved treatment options and who are unable to participate in a clinical trial, to access certain unapproved treatments

  • Sponsor or manufacturer of drug/biologic is responsible for determining whether to make their product available to patients who qualify
  • New statutory requirement for sponsors and manufacturers to provide an annual summary to the FDA


Labeling Changes Regarding Naloxone

Naloxone can be administered by individuals with or without medical training to help reduce opioid overdose deaths

  • Required labeling changes recommend health care professionals prescribe naloxone when they prescribe medicines to treat OUD
  • Also, they consider prescribing naloxone to patients being prescribed opioid pain medicines who are at increased risk of opioid overdose


Image credit: FDA

Market Authorization, Warning: MONJUVI for B-cell lymphoma, TECARTUS for mantle cell lymphoma, EPIDIOLEX for tuberous sclerosis complex, VILTEPSO for duchenne muscular dystrophy, EVRYSDI for spinal muscular atrophy, WARNING for hangover remedies

MONJUVI® (tafasitamab-cxix) for injection

Morphosys Inc

INDICATION: in combination with lenalidomide, is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT).

MECHANISM OF ACTION: Tafasitamab-cxix is an Fc-modified monoclonal antibody that binds to CD19 antigen expressed on the surface of pre-B and mature B lymphocytes and on several B-cell malignancies, including DLBCL, mediates B-cell lysis through apoptosis and immune effector


  • Open label, multicenter single-arm trial, n=81 patients with a diagnosis of DLBCL
  • Endpoint: Overall response rate (ORR), defined as complete and partial responders and response duration
  • Best ORR : 55% (95% CI: 43%, 67%), complete responses in 37%, partial responses in 18%, median response duration was 21.7 months (range: 0, 24)

SAFETY: The most common adverse reactions (≥20%) were neutropenia, fatigue, anemia, diarrhea, thrombocytopenia, cough, pyrexia, peripheral edema, respiratory tract infection, and decreased appetite.


  • Accelerated approval based on ORR, one month ahead of the FDA goal date.
  • Continued approval contingent upon verification and description of clinical benefit in a confirmatory trial(s)
  • Review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment
  • Priority review, fast track, breakthrough, and orphan product designation


TECARTUS™(brexucabtagene autoleucel) suspension for
intravenous infusion

Kite Pharma

INDICATION: CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL)

MECHANISM OF ACTION: Binds to CD19-expressing cancer cells, activate downstream signaling cascades that lead to T cell activation, proliferation, acquisition of effector functions , and
secretion of inflammatory cytokines and chemokines and to killing of CD19-expressing cells


  • Open-label, multicenter, single-arm trial, n=74 patients with relapsed or refractory MCL with prior therapy
  • Primary efficacy outcome measure: Objective response rate (ORR) based on a minimum duration of follow-up for response of six months
  • ORR was 87% (95% CI: 75, 94), with a complete remission (CR) rate of 62% (95% CI: 48, 74)
  • Of all 74 leukapheresed patients, ORR was 80% (95% CI: 69, 88) with a CR rate of 55% (95% CI: 43, 67)


  • Boxed Warning : Cytokine release syndrome and neurologic toxicities with a with a Risk Evaluation and Mitigation Strategy 
  • Most common Grade 3 or higher reactions: Anemia, neutropenia, thrombocytopenia, hypotension, hypophosphatemia, encephalopathy, leukopenia, hypoxia, pyrexia, hyponatremia, hypertension, infection – pathogen unspecified, pneumonia, hypocalcemia, and lymphopenia


  • Accelerated approval based on ORR and durability of response; continued approval contingent upon verification and description of clinical benefit in confirmatory trial
  • Orphan drug designation, breakthrough therapy designation, and priority review


Epidiolex (cannabidiol) [CBD] oral solution 

Greenwich Biosciences Inc.

INDICATION: Treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older

ADDRESSING UNMET NEED: Only FDA-approved drug that contains a purified drug substance derived from cannabis. It is also the second FDA approval of a drug for the treatment of seizures associated with TSC

  • TSC is a rare genetic disease causing non-cancerous (benign) tumors to grow in the brain and other parts of the body like the eyes, heart, kidneys, lungs, and skin
  • Affects about 1 in 6,000 people

MECHANISM OF ACTION: CBD is a chemical component of the Cannabis sativa plant. However, CBD does not cause intoxication or euphoria (the “high”) that comes from tetrahydrocannabinol (THC). Precise mechanism of anticonvulsant effect is unknown


  • Randomized, double-blind, placebo-controlled trial, n=224
  • Primary endpoint: Change from baseline in seizure frequency
  • Significantly greater reduction in the frequency of seizures with Epidiolex
  • Effect seen within eight weeks; consistent throughout the 16-week treatment period
  • Dispensed with patient Medication Guide with drug’s uses and risks
  • Most serious risks: Increase in suicidal thoughts and behavior, or thoughts of self-harm
  • Most common side effects: diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting
  • Additional side effects: liver injury, decreased weight, anemia, and increased creatinine


  • FDA supporting rigorous scientific research on the potential medical uses of cannabis-derived products and working with product developers who are interested in bringing patients safe and effective, high quality products
  • Previously approved for treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS)
  • Not a controlled substance 


VILTEPSO (viltolarsen) injection

NS Pharma

INDICATION: Treatment of Duchenne Muscular Dystrophy (DMD) in patients who
have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping

ADDRESSING UNMET NEED: DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness caused by mutations in the DMD gene that results in an absence of dystrophin. First symptoms are usually seen between three and five years of age and worsen over time. Occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females.

MECHANISM OF ACTION: Binds to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing. Exon 53 skipping allows production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping


  • 2 clinical studies, n=32 patients with genetically confirmed DMD
  • Dystrophin levels increased, on average, from 0.6% of normal at baseline to 5.9% of normal at week 25
  • Increase in dystrophin production is reasonably likely to predict clinical benefit
  • Most common side effects: Upper respiratory tract infection, injection site reaction, cough and fever; kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides- function should be monitored


  • Accelerated approval; based on an increase in dystrophin production in skeletal muscle -continued approval contingent upon verification and description of clinical benefit


EVRYSDI™ (risdiplam) for oral solution


INDICATION: Treatment of Spinal Muscular Atrophy (SMA) in patients 2 months
of age and older

ADDRESSING UNMET NEED: SMA is a rare and often fatal genetic disease affecting muscle strength and movement; second drug and the first oral drug approved to treat disease

MECHANISM OF ACTION: Increases exon 7 inclusion in survival of motor neuron 2 (SMN2) messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain


  • Open-label, infantile-onset SMA study, n= 21 patients, average age 6.7 mo., 12 mo. treatment
  • Efficacy endpoint: Ability to sit without support for at least 5 sec and survival without permanent ventilation vs. natural progression of the disease
  • Meaningful improvement with 41% patients able to sit independently, 81% of patients were alive without permanent ventilation
  • Second randomized, placebo-controlled study, n=180 patients with later-onset SMA, age 2-25 yrs
  • Efficacy endpoint: Change from baseline in MFM32 (a test of motor function) total score at 1 yr; 1.36 increase in score with EVRYSDI vs. 0.19 decrease on placebo
  • Most common side effects: Fever, diarrhea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections
  • Additional side effects for the infantile-onset population: Upper respiratory tract infection, pneumonia, constipation and vomiting


  • Fast track designation, priority review, orphan drug designation,  Rare Pediatric Disease Priority Review Voucher


Hangover Remedies

Warning letters to seven companies whose products claim to cure, treat, mitigate, or prevent hangovers

  • Alcohol intoxication, a poisoning, causes dose-related dysfunction and damage, ranging from mild impairments to death
  • Products from these companies, labeled as dietary supplements, are unapproved new drugs and have not been evaluated by the FDA to be safe and effective for their intended use

Warning letters were sent to the following companies:


Image credits: FDA, Morphosys, Kite, Greenwich, NS Pharma, Genentech

COVID-19 News: BinaxNOW Ag Card + NAVICA app, Advisory Committee to discuss vaccine development, Convalescent plasma, Pooled sample testing and screening, Hand sanitizer warning

BinaxNOW COVID-19 Ag Card & NAVICA App

Abbott Diagnostics

INDICATION: Antigen test where results can be read directly from the testing card to manage risk by quickly identifying infectious people so they don’t spread the disease to others.  Authorized for use in patients suspected of COVID-19 by their healthcare provider within seven days of symptom onset. NAVICA™ app will allow people who test negative to display a temporary digital health pass that is renewed each time a person is tested through their healthcare provider together with the date of the test result

ADDRESSING UNMET NEED: First Diagnostic Test Where Results Can Be Read Directly From Testing Card

  • Highly portable (about the size of a credit card) and provides results in 15 minutes
  • Simple design is fast and efficient for healthcare providers and patients and does not need the use of an analyzer


  • Antigen test using lateral flow technology
  • A healthcare provider swabs the patient’s nose and twirls that sample on a test card with a testing reagent added
  • After waiting 15 minutes, the healthcare provider reads the results directly from the testing card. One line indicates a negative result; two lines indicate a positive result
  • Due to the potential for decreased sensitivity compared to molecular assays, negative results from an antigen test may need to be confirmed with a molecular test prior to making treatment decisions

REGULATORY PATHWAY: Emergency Use Authorization

  • Clinical study with several leading U.S. research universities demonstrated sensitivity of 97.1% (positive percent agreement) and specificity of 98.5% (negative percent agreement) in patients suspected of COVID-19 within the first seven days of symptom onset

 Abbott will sell this test for $5.


Advisory Committee Meeting to Discuss COVID-19 Vaccines

Public meeting of the Vaccines and Related Biological Products Advisory Committee will be held on Oct. 22, 2020

  • To discuss the general matter of the development, authorization, and/or licensure of vaccines indicated to prevent COVID-19
  • Assure that the process and review for vaccine development will be open and transparent
  • Committee made up of outside scientific and public health experts from around the country
  • Public discussion of clinical development of COVID-19 vaccines and data to support safety and effectiveness


VEKLURY (remdesevir)


INDICATION:  Treatment of all hospitalized adult and pediatric patients with suspected or laboratory-confirmed COVID-19, irrespective of their severity of disease.  Specifically, Veklury is only authorized for hospitalized adult and pediatric patients for whom use of an intravenous (IV) agent is clinically appropriate.

MECHANISM OF ACTION: Remdesivir is a nucleoside ribonucleic acid (RNA) polymerase inhibitor


  • One randomized, double-blind, placebo-controlled, n=1,062 hospitalized subjects with mild, moderate and severe COVID-19 who received Veklury (n=541) or placebo (n=521), median time to recovery from COVID-19 was 10 days for the Veklury group compared to 15 days for the placebo group. In hospitalized patients with mild to moderate disease, the results for time to recovery as well as the odds of improvement at Day 15 numerically favored the Veklury group and were consistent with the overall study results.
  • A separate randomized, open-label multi-center trial,  hospitalized adult subjects with moderate COVID-19 treatment with Veklury for five days (n=191) and treatment with Veklury for 10 days (n=193) with standard of care (n=200). Odds of subject’s COVID-19 symptoms improving were statistically significantly higher in the five-day Veklury group at Day 11 when compared to those receiving only standard of care. The odds of improvement with the 10-day treatment group when compared to those receiving only standard of care were numerically favorable, but not statistically significantly different
  • Possible side effects include: increased levels of liver enzymes, which may be a sign of inflammation or damage to cells in the liver; and infusion-related reactions, which may include low blood pressure, nausea, vomiting, sweating, and shivering.



Convalescent Plasma

Convalescent plasma from individuals who have recovered from COVID-19 has not been approved for use by FDA, so it is regulated as an investigational product


Pooled Sample Testing and Screening Testing

FDA encouraging the development of tests for screening asymptomatic individuals and for testing pooled samples

  • Involves mixing several samples together in a “batch” or pooled sample, then testing the pooled sample with a diagnostic test
  • Increases the number of individuals that can be tested using same amount of resources
  • However, because samples are diluted, which could result in less viral genetic material available to detect, there is a greater likelihood of false negative results
  • Works well when there is a low prevalence of cases, meaning more negative results are expected than positive results
  • Several resources being provided


Warning: Hand Sanitizer Packaged in Food and Drink Containers

FDA warning consumers about alcohol-based hand sanitizers that are being packaged in containers that may appear as food or drinks

  • Being packaged in beer cans, children’s food pouches, water bottles, juice bottles and vodka bottles
  • Containing food flavors, such as chocolate or raspberry
  • May put consumers at risk of serious injury or death if ingested


Image credit: Abbott, Gilead, FDA

COVID News: SalivaDirect, Device shortage list, Your digital health, Hand sanitizer warning, Steps to stop spread, Convalescent plasma toolkit


Yale School of Public Health

INTENDED USE & DESCRIPTION: For rapid detection of SARS-CoV-2 using saliva instead of respiratory swabs, which enables non-invasive frequent sampling and reduces the need for trained healthcare professionals during collection.


  • With different combinations of commonly used reagents and instruments, meaning the test could be used broadly in most high-complexity labs
  • Yale intends to provide the SalivaDirect protocol to interested laboratories as an “open source” protocol
  • Because this test does not rely on any proprietary equipment from Yale and can use a variety of commercially available testing components, it can be assembled and used in high-complexity labs throughout the country, provided they comply with the conditions of authorization in the EUA

REGULATORY PATHWAY: Emergency Use Authorization (EUA)

  • Fifth test that uses saliva as a sample for testing


Device Shortage List

FDA monitoring healthcare landscape and supply chain for resulting shortages, or meaningful disruptions to U.S. supply, of certain medical devices

  • Manufacturers of certain devices notify FDA of permanent discontinuance in manufacture of the device
  • Interruption in the manufacture of device that is likely to lead to meaningful disruption in supply during declared public health emergency
  •  Categories of devices in the device shortage list are (1) Personal Protective Equipment (2) Testing Supplies and Equipment (3) Ventilation-Related Products

Device shortage and discontinuance list

FDA Insights: Your Digital Health

Discission on telemedicine, digital health tools, and FDA’s new Digital Health Center of Excellence

Listen , Digital Health page

Hand Sanitizers Consumers Should Not Use

Hand sanitizer products labeled to contain ethanol or isopropyl alcohol but have tested positive for 1-propanol and methanol contamination

  • Young children, adolescents and adults who drink these products as an alcohol (ethanol) substitute are at risk
  • Can cause central nervous system (CNS) depression, which can result in death
  • Exposure can include confusion, decreased consciousness, and slowed pulse and breathing
  • Skin or eye exposure can result in irritation, and rare cases of allergic skin reactions have been reported.


Help Stop the Spread of Coronavirus and Protect Your Family

Remain vigilant and take simple steps

  • Wash Your Hands
  • Wear a Mask and Avoid Crowds 
  • Save Personal Protective Equipment for Those on the Front Lines
  • Follow Food Safety Guidelines
  • Donate Blood 
  • If You Have Fully Recovered From COVID-19, Donate Plasma
  • Report Fraudulent Coronavirus Tests, Vaccines, and Treatments


The Fight Is In Us: Convalescent Plasma Fact Sheets and Toolkit

FDA working together with health care organizations and other partners to slow spread and meeting the challenge of COVID-19

Image credits: Yale, FDA

FDA COVID News: Operation Quack Hack for fraud prevention, Test for IgG antibodies from previous infection, Test for screening prople without known infection, Advanced manufacturing for preparedness, Plasma donation, COVID and Cancer website

FDA Protects Patients and Consumers from Fraud During COVID-19

Operation Quack Hack leverages agency expertise and advanced analytics to protect consumers from fraudulent medical products during the COVID-19 pandemic

  • As of June 2020, team has identified more than 700 fraudulent and unproven medical products 
  • Has reviewed thousands of websites, social media posts, and online marketplace listings
  • > 90 warning letters to sellers, more than 150 reports sent to online marketplaces, and more than 250 abuse complaints sent to domain registrars to date.


First Tests to Estimate Patient’s IgG Antibodies from Past SARS-CoV-2 Infection

ADVIA Centaur COV2G and Atellica IM COV2G, Siemens

COVID-19 serology tests to estimate quantity of antibodies present in the individual’s blood

  • Useful tool to evaluate antibodies to a previous COVID-19 infection
  • Still many unknowns about what the presence of SARS-CoV-2 antibodies and potential immunity
  • Patients cautioned against using the results as an indication to stop taking steps to protect themselves and others
  • Instructions for Use

Advanced Manufacturing to Support Public Health Preparedness

Many medical products are still being manufactured using outdated technologies

  • Can increase the risk of shortages, limit flexibility during an emergency, and contribute to high cost
  • Encouraging “advanced manufacturing,” which refers to new and emerging approaches for production of medical technologies
  • Examples include process intensification methods, such as continuous manufacturing, 3D printing can help produce patient-specific medical devices, digital and smart design and manufacturing processes to increase efficiency and reduce uncertainty
  • READ

First Diagnostic Test for Screening of People Without Known or Suspected COVID-19 Infection

LabCorp COVID-19 RT-PCR Test EUA

For testing people who do not have COVID-19 symptoms or who have no reason to suspect COVID-19 infection, and to allow pooled sample testing 

  • Step toward the type of broad screening that may help enable the reopening of schools and workplaces
  • Also test pooled samples containing up to five individual swab specimens collected under observation
  • Help receiving results sooner, while also conserving vital testing supplies
  • Prescription-only and is authorized for human specimen collection either at home or by a health care provider
  • Only health care provider-collected samples may be pooled at this time
  • Test is as accurate in the broader asymptomatic population as it is among people suspected to have COVID-19
  • EUA Summary

If you have recovered from COVID-19, confirmed by a positive test, you’re in a special position to help us fight the virus. Donate plasma now

New Website on Cancer and COVID-19

FDA Oncology Center of Excellence recognizes cancer patients constitute a vulnerable population at risk ; current priorities

  • Expedite oncology product development
  • Recognize that modifications may be required in clinical trials
  • Innovating collection and analysis of real-world evidence on patient outcomes
  •  Process Expanded Access requests for investigational products
  • Anticipate and prevent drug shortages
  • Inform the cancer community

Cancer and COVID-19

Image credits: FDA, Labcorp, Siemens

COVID-19 updates: Test for pooled samples, CTAP, Domestic inspections, FDA Insights podcasts, Laboratory and Manufacturer resources


Quest SARS-CoV-2 rRT-PCR test for pooled samples

Quest Diagnostics

Emergency Use Authorization for first diagnostic test use with pooled samples containing up to four individual swab specimens

  • Sample pooling allows for more people to be tested quickly using fewer testing resources
  • Samples collected from four individuals tested in pool or “batch” using one test, rather 4 tests
  • If pool is positive, each of the samples in that pool are tested again individually
  • Fewer tests are run overall, meaning fewer testing supplies and quicker test particulalry in areas with low prevalence, meaning most results are expected to be negative.

Package insert

Coronavirus Treatment Acceleration Program

The Coronavirus Treatment Acceleration Program (CTAP) to enable FDA to leverage cross-agency scientific resources and expertise to bear on COVID-19 drugs and biologics development and review


Resumption of domestic inspections with new risk assessment system

COVID-19 Advisory Rating system (COVID-19 Advisory Level) to assist in determining when and where it is safest to conduct prioritized domestic inspections

  • Uses real-time data to qualitatively assess the number of COVID-19 cases in a local area based on state and national data
  • Phase of the State, statistics measured at the county level to gauge the current trend and intensity of infection
  •  Goal of restarting on-site inspections during the week of July 20


FDA Insights podcasts

Insight into issues facing the agency – including the COVID-19 pandemic and other emerging topics.


COVID-19 Resources for Laboratories and Manufacturers

Emergency Use Authorizations for Medical Devices

EUA Authorized Serology Test Performance

Independent Evaluations of COVID-19 Serological Tests

Testing Supply Substitution Strategies: Download the slide show file (PPT – 1.5MB) and click Slide Show > From Beginning.

Reporting fraudulent products

Image credit: FDA

Marketing Authorizations: EndeavorRX digital therapy for ADHD, FINTEPLA for Dravet Syndrome, GALLANT defibrillator systems

EndeavorRx digital therapy

Akili Interactive

INDICATION FOR USE: Digital therapeutic indicated to improve attention function as measured by computerbased testing in children ages 8-12 years old with primarily inattentive or combined-type ADHD, who have a demonstrated attention issue. Patients who engage with EndeavorRx demonstrate improvements in a digitally assessed measure Tests of Variables of Attention (TOVA) of sustained and selective attention and may not display benefits in typical behavioral symptoms, such as hyperactivity. EndeavorRx should be considered for use as part of a therapeutic program that may include: clinician-directed therapy, medication, and/or educational programs, which further address symptoms of the disorder.

DEVICE TYPE: Digital therapy device for Attention Deficit Hyperactivity Disorder. Software intended to provide therapy for ADHD or any of its individual symptoms as an adjunct to clinician supervised treatment.

ADDRESSING UNMET NEED: First digital therapeutic intended to improve symptoms associated with ADHD. First game-based therapeutic granted marketing authorization by the FDA for any type of condition. 


  • Multiple studies in >600 children
  • Evaluated demonstrated improvements in attention function, as measured by TOVA, academic performance measures, and other assessment tools
  • No serious adverse events reported
  • Most common adverse events: Frustration, headache, dizziness, emotional reaction, and aggression

REGULATORY PATHWAY: De Novo classification

Reclassification order

FINTEPLA (fenfluramine) oral solution

Zogenix Inc

INDICATION: Treatment of seizures associated with Dravet syndrome in patients 2 years of age and older

MECHANISM OF ACTION: Exact mechanism for treatment of seizures unknown;
fenfluramine and metabolite, norfenfluramine, increase extracellular levels of serotonin and exhibit agonist activity at serotonin 5HT-2 receptors

ADDRESSING UNMET NEED: New therapy for Dravet syndrome- a life-threatening, rare and chronic form of epilepsy that is characterized by severe and unrelenting seizures despite medical treatment


  • Two clinical studies, n=202 subjects 2-18 years old; Fintepla vs placebo
  • Primary endpoint: Change from baseline in the frequency of convulsive seizures
  • Significantly greater reductions in convulsive seizures frequency with Fintepla within 3-4 weeks, remained consistent over the 14- to 15-week treatment periods
  • Boxed warning: Valvular heart disease (VHD) and pulmonary arterial hypertension (PAH)
  • Most common adverse reactions: Decreased appetite; drowsiness, sedation and lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue or lack of energy; ataxia, balance disorder, gait disturbance, blood pressure; drooling, salivary hypersecretion, pyrexia; upper respiratory tract infection; vomiting; decreased weight; risk of falls; and status epilepticus.


  • Priority Review, Orphan Drug Designation
  • Schedule IV controlled substance
    • Restricted drug distribution program, REMS)
    • Special certification to dispense, cardiac monitoring with echocardiograms


GALLANT implantable cardioverter defibrillator (ICD) and cardiac resynchronization therapy defibrillator (CRT-D) devices 


INDICATION FOR USE: For automated treatment of life-threatening ventricular arrhythmias; also indicated to treat symptoms in patients who have congestive heart failure with ventricular dyssynchrony. In addition, dual chamber ICD and CRT-D devices with the AT/AF detection algorithm are indicated in patients with atrial tachyarrhythmias or those patients who are at significant risk of developing atrial tachyarrhythmias.
MR Conditional ICDs and CRT-Ds are conditionally safe for use in the MRI environment when used in a complete MR Conditional system and according to instructions in the MRI-Ready Systems manual. Scanning under different conditions may result in severe patient injury, death or device malfunction.
The myMerlinPulse™ mobile application is indicated for use by patients with supported Abbott Medical implanted heart devices.


  • Implantable Cardioverter Defibrillator (ICD) and Cardiac Resynchronization Therapy Defibrillator (CRT-D) device intended to provide ventricular antitachycardia pacing and ventricular cardioversion/defibrillation; also intended to resynchronize right and left ventricles
  • Pairs with Abbott’s secure myMerlinPulse™, an iOS- and Android™- compatible mobile smartphone app that helps streamline communication between doctors and their patients
  • App provides people with access to data, device performance, and transmission history, which helps them take an active role in their healthcare
  • Through the app, physicians can continuously monitor their patients remotely allowing for identification of asymptomatic episodes as well as patient-triggered transmissions, which can lead to earlier intervention and reduce clinical burden


  • 6.1 million people in US with cardiac arrhythmias, or abnormal heart rhythms
  • ICDs are used to help reduce the risks of life-threatening arrhythmias.
  • CRT-Ds can be used to restore the heart’s natural pattern of beating for patients with heart failure or in situations when the heart’s chambers beat out of sync


Safety Information

Image credits: Akili, Zogenix, Abbott

COVID News: Hydroxychloroquine sulfate/ chloroquine phosphate revocation, Vaccine development guidance, Partnering with EU, Warning against methanol containing hand sanitizers

Revocation of EUA for hydroxychloroquine sulfate (HCQ) and chloroquine phosphate (CQ) to treat COVID-19

FDA believe’s the suggested dosing regimens for CQ and HCQ unlikely to produce an antiviral effect

  • Earlier observations of decreased viral shedding with HCQ or CQ treatment have not been consistently replicated
  • Recent data from a randomized controlled trial showed no difference between HCQ vs. standard of care slone
  • Current U.S. treatment guidelines do not recommend the use of CQ or HCQ outside of a clinical trial; NIH guidelines now recommend against such use outside of clinical trial
  • Recent data from a large randomized controlled trial with HCQ showed no evidence of benefit for mortality or hospital length of stay or need for mechanical ventilation
  • Additionally, the serious cardiac adverse events and other serious side effects cause the risks to outweigh benefits

Frequently Asked Questions on the Revocation of the Emergency Use Authorization for Hydroxychloroquine Sulfate and Chloroquine Phosphate (PDF, 125 KB)


Development and Licensure of COVID-19 vaccine

Recommendations :

  • Using different technologies such as DNA, RNA, protein and viral vectored vaccines
  • Help expedite vaccine development without sacrificing our standards for quality, safety, and efficacy
  • Guidance provides overview of key considerations chemistry, manufacturing and control, nonclinical and clinical data
  • Studies to directly evaluate the ability of the vaccine to protect humans from SARS-CoV-2 infection and/or disease

Final Guidance

Partnering with the European Union and Global Regulators on COVID-19

FDA, European Commission (EC) and European Medicines Agency (EMA) have a multitude of critical scientific and regulatory collaborations for COVID-19 response  

  • 30 technical expert groups, or “clusters”
  • Exchanging information on interpretation of data supporting regulatory decisions
  • Virtual bilateral meetings to review high-priority areas
  • Engagement with global regulators, under the International Coalition of Medicines Regulatory Authorities (ICMRA) forum, which is comprised of 28 regulatory authorities from around the globe
  • International collaborations in medical device work
  • READ

Action to Warn, Protect Consumers from Dangerous Alcohol-Based Hand Sanitizers Containing Methanol

Hand sanitizer products containing methanol, or wood alcohol

  • Often used to create fuel and antifreeze
  • Can be toxic when absorbed through the skin as well as life-threatening when ingested
  • Reported recent adverse events from adults and children including blindness, hospitalizations and death


Webpage for a full list of FDA-tested and recalled hand sanitizers:

Image credit: FDA

COVID-19 update: FDA Commissioner reflections, Innovations to respond, Testing basics, Hospital beds- stretchers-mattresses, Educational resources, Updated scientific FAQs on testing

The COVID-19 Pandemic — Finding Solutions, Applying Lessons Learned

Reflections from 24th FDA Commissioner Dr. Stephen Hahn

  • Inability to control the events that affect our lives
  • History teaches us that crises often lead to accelerated change and innovations and new discoveries
  • FDA continuously innovating and accelerating measures-for food, drug, devices, diagnostics- to respond to pandemic
  • Learnings will influence FDA functioning post-pandemic


Innovation to Respond to COVID-19

Connect Manufacturers and Health Care Entities: Facilitating information-sharing regarding the use of 3D printing and other advanced manufacturing technologies in the context of personal protective equipment (PPE) and other medical device parts

 Collaborations to Promote Real-World Data Analysis : Harness diverse streams of data to understand and respond to COVID-19 with the potential to contribute to the scientific evaluation of potential diagnostics and interventions

Coronavirus Treatment Acceleration Program (CTAP) : Special emergency program to accelerate new treatments to patients as quickly as possible, while at the same time finding out safety and effectiveness.

Guidance Documents to Support Innovators and Researchers : To accelerate development of prevention and treatment options, increase efficiency of initiating new trials with recommendations for clinical trial designs

COVID-19 Testing Basics

FDA information on different categories of tests

  • Diagnostic Test: Can show if you have an active coronavirus infection and should take steps to quarantine or isolate yourself from others. Currently there are two types of diagnostic tests – molecular (RT-PCR) tests that detect the virus’s genetic material, and antigen tests that detect specific proteins on the surface of the virus.
  • Antibody test: Looks for antibodies that are made by the immune system in response to a threat, such as a specific virus. Antibodies can help fight infections. Antibodies can take several days or weeks to develop after you have an infection and may stay in your blood for several weeks after recovery. Because of this, antibody tests should not be used to diagnose an active coronavirus infection. At this time researchers do not know if the presence of antibodies means that you are immune to the coronavirus in the future.


COVID-19: Hospital Beds, Stretchers, and Mattresses

Information to manufacturers of hospital beds, stretchers, and mattresses to meet increased demand

  • How to know of potential shortage in advance or reporting shortages
  • How does FDA regulate hospital beds, stretchers, and mattresses and what are the guidances
  • What are applicable recognized consensus standards
  • Claims of antimicrobial or antiviral properties
  • Requirements regarding new and non-traditional supply chain partners


COVID-19 Educational Resources

FAQs on Testing for SARS-CoV-2

Image credit: FDA

COVID-19 update: Product terminology, First Standalone at-home sample collection kit , COVID MyStudies App, Guidance to streamline test development, Cancer doctor’s perspective: hydroxychloroquine and chloroquine, Updated EUAs

FDA Terminology of COVID-19 Potential Preventions and Treatments

  • FDA Approved: Independent scientific reviews to determine safety and effectiveness for its intended use and manufactured according to federal quality standards
  • Investigational Treatment:  Experimental- to study use in diease/condition, safety, dose, benefits and risks
  • Expanded Access: Potential pathway for patient with serious/immediately life threatening disease to gain access to investigational product outside of clinical trials
  • Emergency Use Authorization (EUA): Different than approval or clearance; product availability based on the best available evidence, without waiting for all the evidence. Effective until emergency declaration ends

First Standalone At-Home Sample Collection Kit 

Everlywell COVID-19 Test Home Collection Kit

INDICATION: For use by individuals to self-collect nasal swab specimens at home, when determined by a healthcare provider to be appropriate based on results of a COVID-19 questionnaire, and for use only with in vitro diagnostic (IVD) molecular tests for the detection of
SARS-CoV-2 RNA that are indicated for use with the Everlywell COVID-19 Home Collection Kit


  • Used by individuals at home screened via online questionnaire & reviewed by health care provider
  • Self-collect a nasal sample at home using Everlywell’s authorized kit (nasal swabs and tube filled with saline to transport the sample back to a specified lab)
  • Samples tested by specific CLIA-certified laboratories each with individual EUAs to test Everlywell at-home sample collection kit (Fulgent Therapeutics, Assurance Scientific Laboratories)
  • Results to patient through Everlywell’s independent physician network and online portal


  • Data from studies supported by The Bill and Melinda Gates Foundation and UnitedHealth Group to demonstrate stability of specimens during shipping
  • Study data freely available to support other EUA requests, alleviating burden of recreating the same study 


COVID MyStudies App

  • Free platform to remotely obtain informed consent from patients in COVID-19 trials
  • To assist with clinical trials when patients are in or unable to outpatient clinics
  • In Apple App and Google Play


Guidance on Steps to Streamline Development of Tests

  • Steps to further support development of COVID-19 tests for at-home self-collection
  • Voluntary  EUA Template for at-home sample collection kits
  • Template reflects FDA’s current thinking on data and information that developers should submit to facilitate EUA process
  • Includes recommendations for use by laboratories and commercial manufacturers
  • Applies to EUA as well as part of an Institutional Review Board (IRB)-approved study


Cancer Doctor’s Perspective: Hydroxychloroquine and Chloroquine

FDA plays pivotal role to provide new medical products to patients as soon as possible, while at the same time determining effectiveness and if benefits outweigh the risks

Drugs hydroxychloroquine and chloroquine have received particular attention

  • Both are FDA-approved to treat or prevent malaria, and hydroxychloroquine is also approved to treat autoimmune conditions such as chronic discoid lupus erythematosus, systemic lupus erythematosus in adults, and rheumatoid arthritis – have a well-established safety profile for these conditions
  • NOT approved to prevent or treat COVID-19 – subject of numerous clinical trials
  • FDA neither endorses individual prescribing decisions nor prohibits physicians from prescribing
  • It is important that patients and health care providers understand side effects including serious and potentially life-threatening heart rhythm problems as noted in a recent Drug Safety Communication
  • FDA issued EUA that allowed for treatment of certain hospitalized COVID-19 patients for whom a clinical trial is not available, or participation is not feasible
  • Will continue to look at the data to make future determinations on these products based on available evidence including ongoing clinical studies


Updated page on current and terminated Emergency Use Authorizations for COVID-19

Image credit: FDA

COVID-19 updates: Facilitating development and availability of medical products, New actions to accelerate development of novel treatment options, Surveillance inspections


Facilitating development and availability of medical devices

Working to facilitate development and availability of medical products for patients, physicians, healthcare systems as expeditiously and safely as possible

  • Emergency Use Authorizations (EUAs): Quickly allowing unapproved medical products to reach patients in need when there are no adequate, FDA-approved alternatives
  • Deal with supply issues
  • Updated website


Facilitating development and availability of drugs 

Coronavirus Treatment Acceleration Program (CTAP) to expedite the development of potential COVID-19 therapies

  • Several therapies currently being tested in clinical trials to evaluate whether
    they are safe and effective in combating COVID-19
  • National effort to facilitate the development of, and access to, investigational therapies derived from human blood
  • Make treatment options available to patients and providers who are not able to participate in clinical trials through expanded access under investigational drug (IND) applications


New Actions to Accelerate Development of Novel Prevention, Treatment Options for COVID-19

> 130 clinical trials of potential COVID-19 related treatments and additional development programs for other agents are in the planning stages

  •  FDA has started to receive data from clinical studies and expects to receive more
  • Agency intends to engage with pharmaceutical sponsors and other government partners for expedited patient access when favorable results

2 Guidances


Surveillance inspections during COVID-19

CDC collaboration to develop a process to return to on-site facility surveillance inspections in accordance with White House Guidelines for ‘Opening Up America Again’

  • First protect health and well-being of workforce and state contract inspectors as well as industry  health of workers
  • Communications with industry as well as domestic and foreign regulatory counterparts
  • Leadership team meet daily to discuss the urgent issues related to drugs/devices, medical and food product supply chains, leverage public health tools
  • Closely monitor global situation and remain in contact with domestic and foreign regulatory counterparts




COVID-19 Updates: First Antigen Test for rapid detection, First Diagnostic Test using at-home saliva collection, Documents for Reopening, Updated COVID-19 resources


Sofia 2 SARS Antigen FIA
First Antigen Test to Help in Rapid Detection of COVID-19 virus

Quidel Corporation

INDICATION: Qualitative detection of the nucleocapsid protein antigen from SARS-CoV-2 in nasopharyngeal (NP) and nasal (NS) swab specimens directly


  • New category of tests for use in ongoing pandemic
  • Quick detection of protein fragments found on or within the virus


  • Lateral flow immunofluorescent sandwich assay for the qualitative detection of nucleocapsid protein antigen from SARSCoV-2 in nasopharyngeal (NP) and nasal (NS) swab specimens
  • SARS-CoV-2 nucleocapsid protein antigen is generally detectable in respiratory specimens during the acute phase of infection
  • Positive results are indicative of presence of SARS-CoV-2 nucleocapsid protein antigen, but clinical correlation with patient history and other diagnostic information is necessary to determine infection status
  • Negative results should be treated as presumptive and confirmed with a molecular assay

REGULATORY PATHWAY: Emergency Use Authorization


First Diagnostic Test Using At-Home Collection of Saliva Specimens

TaqPath SARS-CoV-2 Assay

Rutgers Clinical Genomics Laboratory

Spectrum Solutions LLC SDNA-1000 Saliva Collection Device

INDICATION: Qualitative detection of nucleic acid from SARS-CoV-2 in oropharyngeal (throat) swab, nasopharyngeal swab, anterior nasal swab, mid-turbinate nasal swab, and bronchoalveolar lavage (BAL) fluid from individuals.  This test is for use with saliva specimens that are self-collected at home with the LLC SDNA -1000 collection device

ADDRESSING UNMET NEED: First diagnostic test where nasal specimens can be collected at home


  • Qualitative test for detection of nucleic acid from SARS-CoV-2
  • Self-collection saliva specimens at home or in a healthcare setting
  • SARS-CoV-2 nucleic acid generally detectable in respiratory specimens during the acute phase of infection
  • Positive results are indicative of the presence of SARS-CoV-2 nucleic acid; clinical correlation with patient history and other diagnostic information is necessary to
    determine patient infection status
  • Positive results do not rule out bacterial infection or coinfection with other viruses

REGULATORY PATHWAY: Emergency Use Authorization



Documents to support Reopening

FDA issued documents designed to assist retail food establishments in preparing to reopen

  • Help businesses that prepare food to serve or sell to the public directly, such as restaurants, bakeries, bars and carry-outs, protect employee and public health as they reopen for business

Food Safety Checklist


Updated COVID-19 Resources

FAQs on Testing for SARS-CoV-2

FDA COVID-19 Response: At-A-Glance Summary

Image credit: FDA

COVID-19 Updates: Remdesivir, Convalescent plasma, Tips & FAQs (EUAs, Alcohol-based hand sanitizer, Medical Countermeasures Initiative, Grocery shopping, Pet safety


REMDESIVIR injection


AUTHORIZED USE: Treatment of patients hospitalized with suspected or laboratory confirmed SARS-CoV-2 infection and severe disease.

Severe disease is defined as patients with an oxygen saturation (SpO2) ≤94% on room air or requiring supplemental oxygen or requiring mechanical ventilation or requiring extracorporeal membrane oxygenation (ECMO). Specifically, remdesivir is only authorized for hospitalized adult and pediatric patients for whom use of an intravenous agent is clinically appropriate.

MECHANISM OF ACTION: Nucleoside ribonucleic acid (RNA) polymerase inhibitor that inhibits viral RNA synthesis; activity in cell culture and animal models against SARS-CoV, MERS-CoV, and SARS-CoV-2


NIAID-sponsored randomized, double-blinded, placebo-controlled trial

  • Randomized, double-blind, placebo-control, remdesivir vs. placebo, n= 1063 hospitalized patients
  • Primary clinical endpoint: Time to recovery within 28 days after randomization
  • Preliminary analysis:  Median time to recovery was 11 days (remdesivir) vs. 15 days (placebo) p<0.001), Mortality was 8.0% (remdesivir) vs. 11.6% (placebo), p=0.05

Gilead-sponsored open-label trial

  • Randomized, open-label multi-center clinical trial, n~400 with severe COVID-19 with 5-day or 10-day treatment
  • Primary clinical endpoint: Clinical status assessed by 7-point ordinal scale at Day 14 after randomization
  • Preliminary Analysis: Similar improvement in clinical status in both groups


  • Hepatic adverse reactions: Transaminase elevations, ALT and AST, need for appropriate clinical and laboratory monitoring

REGULATORY PATHWAY: Emergency Use Authorization

  • SARS-CoV-2 can cause  serious or life-threatening disease or condition
  • Based on the totality of scientific evidence available to FDA, it is reasonable to believe that remdesivir may be effective in treating COVID-19
  • There is no adequate, approved, and available alternative to the emergency use of
    remdesivir for the treatment of COVID-19



Investigational COVID-19 convalescent plasma

Providing  recommendations to health care providers and investigators on the administration and study of investigational convalescent plasma

Donor eligibility

  • Evidence of COVID-19 documented by a laboratory test or positive serological test
  • Complete resolution of symptoms at least 14 days before donation
  • SARS-CoV-2 neutralizing antibody titers, if available

Patient eligibility

  • Laboratory confirmed COVID-19
  • Severe or immediately life-threatening COVID-19
  • Informed consent



COVID-19 Tips and FAQs

What is Emergency Use Authorization

Updated EUA Webpage

Availability of alcohol-based Hand sanitizer

Medical Countermeasures Initiative (MCMi)

12 tips for grocery shopping

Pet safety

Image credit: Gilead, FDA

COVID-19 Updates: At-a-glance resource, Caution on Hydroxychloroquine or Chloroquine use, Remote digital pathology devices


FDA COVID-19 RESPONSE: At-a-Glance Summary

Major focus areas

  • Testing, therapeutics, and devices such as ventilators and personal protective equipment
  • Vaccine development
  • Food supply
  • Action on fraudulent products


Cautions against use of hydroxychloroquine or chloroquine for COVID-19 due to risk of heart rhythm problems

  • Reports of serious and potentially life-threatening heart rhythm problems
  • May be mitigated when health care professionals closely screen and supervise COVID-19 patients in a hospital setting or a clinical trial


Remote Digital Pathology Devices During COVID-19 Emergency

Guidance to help expand the availability of digital pathology devices for remote reviewing and reporting of scanned digital images of pathology slides during this pandemic

  • Automated Digital Image Manual Interpretation Microscope
  •  Whole Slide Imaging System
  • Digital Pathology Image Viewing and Management Software
  • Digital Pathology Display

Expanded, off-label use for the duration of the COVID-19 public health emergency

  • Facilitate remote reviewing and reporting of pathology slides
  • Help facilitate continuity of patient care
  • Reduce healthcare personnel contact and risk of exposure


Image credit: FDA

COVID-19 Updates


ACTIV Public-private partnership to speed COVID-19 vaccine and treatment options

NIH announced ‘Accelerating COVID-19 Therapeutic Interventions and Vaccines’ (ACTIV) partnership with FDA and others to speed the development of COVID-19 vaccine and treatment options

  • Standardize and share preclinical evaluation methods in open forum allowing for comparison and validation
  • Prioritize and accelerate clinical evaluation of therapeutic candidates with near-term potential
  • Maximize clinical trial capacity and effectiveness
  • Advance vaccine development


Protecting Public Health from Fraudulent and Potentially Harmful Products

Temporary injunction against the Genesis II Church of Health and Healing (Genesis)

  • Unlawfully distributing MMS: When combined with included activator, has a chlorine dioxide content equivalent to industrial bleach
  • There is danger that defendants will continue violating the law without temporary restraining order; defied previous Warning letter

Warning letter to Nova Botanix LTD DBA CanaBD

  • Selling unapproved and misbranded cannabidiol (CBD) product deliberately misleading claims for prevention and treatment of COVID-19
  • Currently NO FDA-approved products to prevent or treat COVID-19

Expansion of COVID-19 Testing by Using Synthetic Swabs 

FDA, Gates Foundation, UnitedHealth Group, Quantigen, U.S. Cotton Collaborate to Address Testing Supply Needs

Synthetic swabs – with a design similar to Q-tips – could be used to test patients by collecting a sample from the front of the nose

U.S. Cotton

  • Developed polyester-based, synthetic Q-tip-type swab  for compatibility with COVID-19 testing
  • Plans to produce the new swabs in large quantities for increased diagnostic testing

UnitedHealth Group, Quantigen, Gates Foundation

  • Clinical investigation to support synthetic swab use
  • Testing to support swabbing at  front of nose used enabling more comfortable, self-collection by patients and limiting exposure of healthcare providers


Emergency Use Authorization (EUA): Seraph 100 Microbind Affinity Blood Filter device

INDICATION FOR USE: Extracorporeal blood purification (EBP) device) to treat patients 18 years of age or older with confirmed COVID-19 admitted to the ICU with confirmed or imminent respiratory failure to reduce pathogens and inflammatory mediators from the bloodstream

  • Designed to reduce bacteria, viruses, toxins, cytokines and other inflammatory mediators from whole blood
  • Form factor very similar to other blood filters, such as hemodialyzers or hemoperfusion filters – compatible with hemodialysis systems that use industry standard bloodline connectors for ease of operation, training, and utility


COVID-19 Diagnostics Update


FDA COVID-19 actions


FDA COVID-19 updates

The Agency is playing an integral role in the coronavirus fight, using science and innovative approaches as well as joining in the healthcare frontlines

Here are some of the recent actions

Coronavirus Treatment Acceleration Program (CTAP)


Coronavirus Treatment Acceleration Program (CTAP)

 Special emergency program for possible therapies – to be updated real time


  • 10 therapeutic agents in active trials
  • Another 15 therapeutic agents in planning stages


  • Rapid responses to queries, ultra-rapid review of protocol, development plans
  • Review single patient expanded access requests within 3 hours



COVID-19 Emergency Use Authorizations, Diagnostic Testing policy, N95 Respirators and Surgical Masks


COVID-19 Emergency Use Authorizations (EUA) for Medical Devices and Diagnostics

EUA being granted as there is significant potential for public health emergency that can affect national security or the health and security 

  1. Personal Protective equipment
  2. Diagnostic tests

Personal Protective Equipment


  • Disposable filtering facepiece respirators (FFRs) approved by the National Institute for Occupational Safety and Health (NIOSH)
  • FFRs that were NIOSH-approved but have since passed the manufacturers’ recommended shelf-life, for use in healthcare settings by healthcare personnel (HCP) to prevent wearer exposure to pathogenic biological airborne particulates

Diagnostic Tests


  • Qualitative Real-Time RT-PCR test for qualitative detection of nucleic acid from SARS-CoV-2 in upper and lower respiratory specimens from individuals suspected of COVID-19 by their healthcare provider
  • Positive test result  indicates RNA detected, patient is infected and presumed to be contagious; patient management should follow current CDC guidelines
  • Negative test result means RNA was not present in specimen; does not rule out COVID-19 and should not be used as the sole basis for treatment or patient management decisions




Policy for Diagnostic Tests for COVID-19 during the Public Health Emergency 

Two policies for accelerating the development of certain laboratory tests for COVID-19

  1. EUA submission to FDA
  2. No EUA submission
    • When test is developed under State authorities and State takes responsibility for testing by laboratories
    • CLIA certified and meet requirements

Laboratories offering testing under Policy

  • AdventHealth
  • ARUP Laboratories
  • Assurance Scientific
  • Baylor Scott and White Medical Center – Temple
  • BioReference Laboratories
  • The Children’s Hospital of Philadelphia
  • Diatherix Eurofins
  • Emory Medical Laboratory, Emory Healthcare
  • Gravity Diagnostics
  • Henry Ford Health System
  • HMH Hackensack University Medical Center
  • Hospital of the University of Pennsylvania
  • Houston Methodist Hospital
  • Integrity Laboratories
  • Johns Hopkins Medical Microbiology Laboratory at Johns Hopkins Hospital
  • Montefiore Medical Center
  • New York Presbyterian Hospital -Weill Cornell Medicine (NYPH-WCM)
  • Next Bio-Research Services LLC
  • NYU Langone Medical Center
  • Quest Diagnostics Infectious Disease, Inc.
  • Stanford Health Care Clinical Laboratory
  • Texas Children’s Hospital Department of Pathology
  • TGen North, Clinical Laboratory
  • UCSF-Health
  • University of Washington
  • Viracor Eurofins Clinical Diagnostics

Other FAQs


N95 Respirators and Surgical Masks (Face Masks)

FDA regulates surgical masks and surgical N95 respirators differently based on their intended use

  • Surgical mask : Loose-fitting, disposable device that creates physical barrier between mouth and nose of wearer and potential contaminants in the immediate environment. Edges of mask are not designed to form seal around nose and mouth
  • N95 respirator: Respiratory protective device designed to achieve very close facial fit and very efficient filtration of airborne particles; edges designed to form seal around the nose and mouth.

N95 Respirators not for public use 


Image credit: FDA, CDC

COVID-19 News: New expedited diagnostic testing policy, New York SARS-CoV-2 diagnostic authorization, Medical product shortages, Misinformation about natural products


Policy for COVID-19 Diagnostics Testing in Laboratories Certified to Perform High Complexity Testing under CLIA prior to Emergency Use Authorization 

Policy for novel COVID-19 molecular diagnostics tests developed and used in CLIA laboratories prior to FDA issuance of emergency use authorizations (EUA) 

  • Public health emergency circumstances requiring rapid diagnostic testing to control emergence
  • Rapid COVID-19 detection with accelerated policy enabling CLIA laboratories for more rapid testing capacity
  • Immediately in Effect guidance
  • Accelerated Emergency Use Authorization Template



New York SARS-CoV-2 Realtime Reverse Transcriptase (RT)-PCR Diagnostic Panel 

Wadsworth Center, New York State Department of Public Health 

INDICATION: Presumptive qualitative detection of nucleic acid from SARS-CoV-2 in  nasopharyngeal/oropharyngeal swabs and sputa collected from individuals who meet CDC COVID-19 clinical and/or epidemiological criteria


  • Human Specimen Control (HSC): A human cell culture preparation used as an extraction control and positive control for the RNase P primer and probe set that is extracted and tested concurrently with each specimen extraction run
  • SARS-CoV-2 Positive Control (SARS-CoV-2 Pos): Run with each batch of specimens. Monitors improper assay setup, reagent failures of rRT-PCR reagents and reaction conditions
  • No Template Control (NTC): Nuclease-free water included in each run. Monitors for reagent and system contamination
  • RNase P (RP) control in clinical samples: The RP primer and probe set is included in each run to test for human RNase P, which controls for specimen quality and demonstrates that nucleic acid was generated by the extraction process

REGULATORY PATHWAY: Emergency Use Authorization

  • SARS-CoV-2 can cause a serious or life-threatening disease or condition
  • Based on the totality of scientific evidence, reasonable to believe diagnostic panel may be effective in diagnosing COVID-19
  • There is no adequate, approved, and available alternative

Letter of Authorization

Fact Sheet


COVID-19 Supply Chain Update

Outbreak would likely impact the medical product supply chain, including potential disruptions to supply or shortages of critical medical products in the U.S

  • Alert from a manufacturer about drug shortage due to disruption in China manufacturing
  • In contact with >180 manufacturers of human drugs
  • In contact with 63 manufacturers of essential medical devices
  • Preparing for increasing demand in personal protective equipment—surgical gowns, gloves, masks, respirator protective devices etc
  • Not aware of any cellular or gene therapies that are made in China
  •  No reports of transmission by food or food packaging

Proposals  to prevent or mitigate medical product shortages

  • Lengthen expiration dates
  • Require risk management plans
  • Improve data sharing and require more accurate supply chain information
  • Establish reporting requirements for device manufacturers




Image credits: FDA, NIH

News & Views: COVID-19 updates, Rare Disease efforts, Fraudulent DTC genetic tests & review, Stopping illicit medical products from India, Updated Insulin regulatory pathway, Directory for approved drugs (since 1985)


FDA’s Actions in Response to 2019 Novel Coronavirus at Home and Abroad

Active partner in Novel Coronavirus (COVID-19) response, working closely with  Department of Health and Human Services, as well as with international counterparts

  • Active Supply Chain Surveillance: Closely monitor domestic and global supply chain
  • Inspections and Monitoring Compliance of FDA Products Manufactured Overseas: Risk-based surveillance testing of imported products, including those from China
  • Safety of Consumer Products: Close monitoring of fraudulent products and false product claims related to COVID-19
  • Efforts to Diagnose, Treat and Prevent: Expedite development and availability of medical products needed to diagnose, treat and prevent disease
  • Next Steps: Collaborating with interagency partners, international partners, medical product developers and manufacturers to help advance response efforts combat this virus



Rare Disease Day 2020: FDA Continues Important Work on Treatments for Rare Diseases

Commemorating  Rare Disease Day by efforts to get treatments to patients with rare diseases across the thousands of identified rare diseases

  • Public meeting with stakeholders to address challenges and opportunities surrounding rare disease product development
  • New request for applications (RFA) for the Orphan Products Grants Program
  • Additional information on orphan “exclusivity protected uses.”
  • Enhanced rare disease patient website




Collaborative Review of Scientific Evidence to Support Associations Between Genetic Information and Specific Medications

Concerns with DTC genetic tests making claims about genetic test results to manage medication treatment that are NOT supported FDA-approved drug labeling or other scientific evidence

  • Steps to help ensure that claims are grounded in sound science to avoid inappropriate management of patients’ medications
  •  New web-based resource including table describing some of the gene-drug interactions for altered drug metabolism, differential therapeutic effects, differences in risks of adverse events
  • Continue to review various professional guidelines e.g. Clinical Pharmacogenetics Implementation Consortium
  • Continue participation in community-based collaborative approach for ongoing evaluation of the evolving science

Table of Pharmacogenetic AssociationsREAD


FDA Takes Action with Indian Government to Protect Consumers From Illicit Medical Products

Operation Broadsword targeted packages entering the U.S. through International Mail Facility from Jan. 28 through Jan. 30

  • Investigators from both governments examined > 800 shipments, ~50 different FDA-regulated products, many transshipped through third-party countries to conceal point of origin and avoid detection
  • Stopped approximately 500 shipments of illicit, and potentially dangerous, unapproved prescription drugs and combination medical devices from reaching American consumers
  • FDA provided consumer information :  BeSafeRx: Know Your Online Pharmacy.  and encouraged to report any unlawful sale of medical products online to FDA.



Smooth Regulatory Transition of Insulin and Other Biological Products  to Increase Patient Access and Potentially Lower Prices on Insulin

Steps to ensure a smooth transition to new regulatory pathway

  • Final Rule for Biosimilars Action Plan
  • Incorporates changes made by the Biologics Price Competition and Innovation Act of 2009 (BPCI Act) and the Further Consolidated Appropriations Act, 2020
  • Intended to balance innovation and competition and facilitate the development and approval of biosimilar and interchangeable product




Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals

New resource to assist external and agency researchers collecting historical information about FDA’s drug approvals 

  • Provide researchers with curated data regarding drug products approved by CDER since 1985 through 2019
  • Facilitate data accessibility, transparency, and accuracy when researchers seek information about an approved drug
  • Derived from FDA internal databases and document records, and reflects the state of each application at the time of initial regulatory approval
  • CDER will manage the compilation and will update it periodically with the latest drug approval data

Compilation of CDER NME and New Biologic Approvals 1985-2019

Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals – Data Dictionary

Image credit: FDA

Market Authorizations: 2019-nCoV diagnostic, ERVEBO Ebola vaccine, TEPEZZA for thyroid eye disease

Capture2019-nCoV Real-Time RT-PCR Diagnostic Panel


INTENDED USE: For the presumptive qualitative detection of nucleic acid from the 2019-nCoV in upper and lower respiratory specimens (such as nasopharyngeal or oropharyngeal swabs, sputum, lower respiratory tract aspirates, bronchoalveolar lavage, and nasopharyngeal wash/aspirate or nasal aspirate) collected from individuals who meet CDC criteria for 2019-nCoV testing. Testing is limited to qualified laboratories
designated by CDC and, in the United States, certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), to perform high complexity tests.




  • Reverse transcriptase polymerase chain reaction (PCR) test that provides presumptive detection of 2019-nCoV from respiratory secretions, such as nasal or oral swabs
  • Positive test result indicates likely infection with 2019-nCoV and infected patients should work with their health care provider to manage their symptoms and determine how to best protect the people around them
  • Negative results do not preclude 2019-nCoV infection and should not be used as the sole basis for treatment or other patient management decisions; negative results must be combined with clinical observations, patient history and epidemiological information

REGULATORY PATHWAY: Emergency Use Authorization to address public health  emergency



ERVEBO ebola vaccine for intramuscular injection


INDICATION: Prevention of disease caused by Zaire ebolavirus in individuals 18
years of age and older

ADDRESSING UNMET NEED: First FDA-approved vaccine for the prevention of Ebola virus disease (EVD), caused by Zaire ebolavirus

MECHANISM OF ACTION: Immunization results in an immune response and protection from disease caused by Zaire ebolavirus


  • Guinea study, during 2014-2016 outbreak, n= 3,537 contacts and contacts of contacts with laboratory-confirmed EVD, “immediate” or 21-day “delayed” vaccination
  • ERVEBO 100% effective in preventing Ebola cases with symptom onset greater than 10 days after vaccination
  • No EVD cases in “immediate” cluster group vs 10 cases in 21-day “delayed” cluster group
  • Liberia study- n= 477, Sierra Leone study – n=500, Canada, Spain, US study – n=900
  • Antibody responses in Canada, Spain and U.S. study were similar to those among individuals in the studies conducted in Liberia and Sierra Leone.


  • Most commonly reported side effects were pain, swelling and redness at the injection site, as well as headache, fever, joint and muscle aches and fatigue.


  • Priority Review and Tropical Disease Priority Review Voucher, Breakthrough Therapy designation
  •  Because of public health importance, FDA worked closely with Merck and completed its BLA evaluation in less than six months



TEPEZZA (teprotumumab-trbw) injection, for intravenous use

Horizon Therapeutics

INDICATION: Treatment of Thyroid Eye Disease


  • First drug approved for the treatment of thyroid eye disease-
  • Rare condition where muscles and fatty tissues behind eye become inflamed, causing eyes to be pushed forward and bulge outwards (proptosis)

MECHANISM OF ACTION: Not been fully characterized- binds to IGF-1R and blocks  activation and signaling


  • Two studies, n=170 patients with active thyroid eye disease, TEPEZZA or placebo
  • Primary endpoint: >2 millimeter reduction in proptosis- 71% (Study 1) and 83% (Study 2) vs. 20% and 10%


  • Most common adverse reactions: Muscle spasm, nausea, alopecia, diarrhea, fatigue, hyperglycemia, hearing loss, dry skin, dysgeusia, headache
  • Should not be used if pregnant, counseling on pregnancy prevention during treatment and for 6 months following last dose


  • Priority Review, in addition to Fast Track and Breakthrough Therapy Designation 
  • Orphan Drug designation, with support from FDA Orphan Products Grants Program
  • Postmarketing commitments: Additional studies to characterize efficacy, safety, quality


Image credits: FDA, Merck, Horizon Therapeutics








News and Views: Coronavirus medical countermeasures, 2019 New Therapies, Gene Therapy innovation, Deter anti-competitive business for biologics, Heart Health education for women, HIV Drugs mobile database, Warning for nicotine toothpicks


Advance Development of Novel Coronavirus Medical Countermeasures (MCM)

FDA collaborating with interagency partners, product developers, international partners and global regulators to
  • Expedite development and availability of MCM to diagnose, treat, mitigate, prevent
  • Utilize pathways, including Emergency Use Authorization (EUA) to more rapidly make MCM available
  • Provide regulatory advice, guidance, and technical assistance to  sponsors developing investigational MCMs
  • Provide review and feedback on development proposals including design and set-up of clinical trials
  • Protect safety of nation’s blood supply and human cells, tissues,cellular/tissue-based products for transplantation
  • Enable access to investigational MCM through EUA or expanded access mechanisms
  • Protect consumers against fraudulent products by monitoring fraudulent products and false product claims and taking appropriate action

READ , 2019-nCOV website 


2019 CDER New Drug Approvals Report

Annual report entitled Advancing Health Through Innovation: New Drug Therapy Approvals

  • Variety of novel drugs for advancing patient care – never approved in US
  • Overview of other notable approvals — new uses of uses for approved drugs
  • Treat new population of patients, such as children
  • Innovative ways to enhance efficiency and expedite review and approval

Innovation and Access areas

  • Rare Diseases
  • Neurological and Psychiatric disorder
  • Infectious diseases
  • Heart, Lung, Circulatory, Endocrine diseases
  • Autoimmune conditions
  • Women’s and Men’s specific health issues
  • Cancers and blood disorders
  • Biosimilars



Strong Support of Innovation in Development of Gene Therapy Products

FDA efforts  to support innovators developing new gene therapy products, which insert new genetic material into a patient’s cells

  • 4 products approved
  • > 900 investigational new drug (IND) applications for ongoing clinical studies
  • will serve to improve therapeutic choices

Publication of guidances issued to provide recommendations for product developers



New Efforts to Further Deter Anti-Competitive Business Practices, Support Competitive Market for Biological Products to Help Americans

FDA and FTC to collaborate to curtail and discourage anti-competitive behavior to faciliate robust competition and bring down cost for biologics

  1. Promote greater competition in biologic markets
  2. Deter behavior that impedes access to samples needed for the development of biologics, including biosimilars
  3. Take appropriate action against false or misleading communications
  4. Review patent settlement agreements involving biologics, including biosimilars, for antitrust violations


CaptureHeart Health Education for Women

FDA Office of Women’s Heath (OWH) initiatives is support of American Heart Month
  • Heart health educational video ‘Getting a Beat : On what women know about heart health
  • New KNOWH the Difference initiative, which focuses on sharing important knowledge and news on women’s health (KNOWH)
  • OWH research and extramural funding opportunities

Mobile-Friendly Database with Information on Life-Saving HIV Drugs

Interactive database  on critical information about antiretrovirals (ARVs) eligible for purchase under the President’s Emergency Plan for AIDS Relief (PEPFAR) program

  • Empower public and health care providers by enhancing the amount and availability of information and data provided on each drug
  • Health care providers, consumers, procurer access to FDA-reviewed product labeling and essential scientific information for safe and effective use of drug
  • Information on pediatric drugs, where manufactured, shelf-life, storage requirements
  • Ability to export reports, collect metrics, readily access on mobile platforms


FDA Warns Maker of Nicotine-Containing Toothpicks of Several Violations, Including Illegal Sales 

  • Warning letter to Smart Toothpicks LLC,  Tempe, Arizona, for selling dissolvable tobacco products, including Peppermint Ice Nicotine Toothpicks
  • Three specific violations:
    • selling a tobacco product to a minor through the company’s website
    • selling unauthorized modified risk tobacco products
    • failing to include required nicotine warning statements on both packaging and advertising
  • Written response to this letter within 15 working days on corrective actions
    • discontinue violative labeling, advertising, sale, and/or distribution
    • plan for maintaining compliance with the FD&C Act

Action is part of the agency’s Youth Tobacco Prevention Plan, to prevent and reduce youth tobacco use 


Image credits: FDA

Device Authorizations: COBAS MRSA diagnostic, WOUNDCHECK for bacterial status, GSP Neonatal Creatine Kinase for Duchenne, CONTROL-IQ for diabetes

CaptureCOBAS vivoDx MRSA diagnostic test 

Roche Molecular Systems Inc.

INDICATION FOR USE: Automated qualitative in vitro diagnostic test for the direct detection of live methicillin-resistant Staphylococcus aureus (MRSA) cells in nasal swab samples from patients who are at risk for nasal colonization by MRSA.
The test utilizes selective agents and bioparticles (Smarticles technology) to introduce a luciferase gene into targeted bacteria to create an amplified luminescent signal in only viable (live) MRSA cells. The cobas vivoDx MRSA test is intended to aid in the prevention and control of MRSA infections in healthcare settings. It is not intended to diagnose MRSA infections, nor to guide, or monitor treatment. Concomitant cultures are necessary to recover organisms for epidemiological typing or for further susceptibility testing.

ADDRESSING UNMET NEED: New tool in the fight to prevent and control MRSA in high-risk settings

DESCRIPTION: System for detection of microorganisms and antimicrobial resistance using reporter expression

  • New bacteriophage technology based on bioluminescence to detect MRSA from nasal swab samples in 5 hours vs  compared to 24-48 hours for conventional culture

PERFORMANCE TESTING: Test correctly identified MRSA in  90% of samples where MRSA was present and 98.6% of samples with no MRSA

RISKS: Failure to use the device correctly, False positive or negative results, Failure to interpret results correctly

MITIGATIONS: Labeling, Design verification and validation, Other special controls

REGULATORY PATHWAY: De Novo premarket review pathway



WOUNDCHEK Bacterial Status

Woundcheck Laboratories

INDICATION FOR USE: in vitro diagnostic chromatographic test for qualitative detection of bacterial protease activity directly from wound fluid samples collected with a swab. The WCBS test is intended for use in adult patients as an aid in assessing the risk for nonhealing of chronic venous, diabetic foot, and pressure ulcers associated with wounds where there are no signs of wound infection and where patients are asymptomatic for clinical signs of infection. The test is intended for use with chronic wounds that are between 21 days and < 6 months of age and chronic wounds that are ≥ 6 months of age that are < 1cm2 in size.

DEVICE TYPE: To detect bacterial protease activity in chronic wound fluid

  • Lateral flow prescription device that may include a sterile swab
  • Intended for use in patients as an aid in assessing the risk for non-healing of chronic venous, diabetic foot, and pressure ulcers associated with wounds where there are no signs of wound infection and where patients are asymptomatic for clinical signs of infection.

RISKS: Failure to use the device correctly, False positive or negative results, Failure to interpret results correctly

MITIGATIONS: Labeling, Design verification and validation, Other special controls

REGULATORY PATHWAY: De Novo premarket review pathway



GSP Neonatal Creatine Kinase – MM kit


ADDRESSING UNMET NEED: First test to aid in newborn screening for Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness

DESCRIPTION: Muscular dystrophy newborn screening test

  • Intended to measure creatine kinase levels obtained from dried blood spot specimens on filter paper from newborns (prick of a newborn’s heel 24 to 48 hours after birth) as an aid in screening newborns for muscular dystrophy
  • Kit works by measuring the concentration of a type of protein called CK-MM, which is part of a group of proteins called creatine kinase
  • Creatine kinase is found in muscle tissue and CK-MM enters the blood stream in increased amounts when there is muscle damage


  • Clinical study, n=3,041 newborns, dried blood samples tested for DMD associated protein levels
  • Kit able to accurately identify the four screened newborns that had DMD-causing genetic mutations
  • Additional testing of 30 samples from newborns with clinically confirmed cases of DMD; all correctly identified

RISKS: False positive and False negative results

MITIGATIONS: Labeling, Design verification and validation, Special controls

REGULATORY PATHWAY: De Novo premarket review pathway


CaptureControl-IQ Technology

Tandem Diabetes Care

INDICATION FOR USE: Intended for use with compatible integrated continuous glucose monitors (iCGM) and alternate controller enabled (ACE) pumps to automatically increase, decrease, and suspend delivery of basal insulin based on iCGM readings and predicted glucose values. It can also deliver correction boluses when the glucose value is predicted to exceed a predefined threshold.
Control-IQ technology is intended for the management of Type 1 diabetes mellitus in persons 14 years of age and greater.
Control-IQ technology is intended for single patient use and requires a prescription.
Control-IQ technology is indicated for use with NovoLog or Humalog U-100 insulin.

DESCRIPTION: Interoperable automated glycemic controller

  • Intended to automatically calculate drug doses based on inputs such as glucose and other relevant physiological parameters, and to command the delivery of such drug doses from a connected infusion pump
  • Designed to reliably and securely communicate with digitally connected devices to allow drug delivery commands to be sent, received, executed, and confirmed. Interoperable automated glycemic controllers are intended to be used in
    conjunction with digitally connected devices for the purpose of maintaining glycemic control.


  • Inability of the controller to handle different pharmacokinetic and pharmacodynamic characteristics of the drugs
  • Lack of compatibility of connected devices
  • Connected devices having inadequate performance to allow safe use of  controller
  • Failure to report device malfunctions or adverse events to manufacturer
  • Latent flaws in software
  • Failure to provide appropriate treatment due to loss of communication with
    connected devices
  • Risk due to insecure transmission of data
  • Failure to correctly determine the root cause of device malfunctions
  • Data transmission interference/electromagnetic disturbance

MITIGATIONS: Device performance, drug compatibility information in labeling
User training, Validation of processes, and procedures as well as limitations with connected and interoperable devices, Assigning post-market responsibilities. Software validation testing, Electrical safety verification and validation, electromagnetic compatibility,radio frequency wireless testing, user training

REGULATORY PATHWAY: De Novo premarket review pathway


Image credits:  Roche, Woundcheck Labs, PerkinElmer, Tandem

News & Views: 24th FDA Commissioner, Drug importation from Canada, Center of Excellence for drug compounding, Acute pain opioid prescribing, Generic drug pricing, Operation Vapor Lock, Naloxone and opioid overdose, 2019-2020 Flu season



Stephen Hahn, MD, confirmed as the 24th FDA Commissioner

  • Radiation Oncologist with Residency at the National Cancer Institute
  • Professor at University of Pennsylvania
  • Chief Medical Executive at MD Anderson Cancer Center


Steps to lower U.S. prescription drug prices

Allow importation of certain prescription drugs shipped from Canada

  • Purpose of proposed rule is to lower prices and reduce out of pocket costs
  • Foreign seller, licensed by Health Canada and registered with FDA, to purchase directly from manufacturer
  • US importer, subject to the supply chain security requirements, to buy directly from foreign seller
  • Importer arranges for statutorily prescribed testing for authenticity, degradation, and other requirements by a qualifying US laboratory
  • Post-importation requirements including adverse event, medication error, field alert to manufacturer and to FDA


CaptureImproving quality of compounded drugs

Novel approaches to reduce risks with production practices of outsourcing facilities 

  • Establishing Compounding Quality Center of Excellence to enhance collaboration among and provide educational programs for outsourcing facilities
  • Three main areas of focus: in-person, online education and trainings, conference to exchange ideas and best practices, market research help inform FDA on key issues


Capture.JPGCapture.JPGNational Academies of Sciences, Engineering, and Medicine (NASEM) report on framing opioid prescribing guidelines for acute pain

FDA contracted NASEM for evidence-based guidelines for opioid analgesic prescribing for acute pain. NASEM recommendations are:

  • Develop an analytic framework (e.g., Figure above ) to develop and assess evidence base for clinical practice guidelines (CPG)
  • Outpatient opioid prescribing CPGs should explicitly state patient populations  (e.g., adults versus children) and contextual aspects (e.g. setting, prescriber type, prior treatments)
  • To determine optimal opioid prescribing strategies, examine not only intermediate outcomes (e.g. pills prescribed, unused, long-term opioid use), but also the short- and long-term health outcomes (e.g. mortality, overdose, opioid use disorder, pain, and function)
  • Well-designed observational and quality improvement initiatives helpful for evaluating the effects of opioid prescribing strategies on health outcomes
  • CPGs should be implemented by governmental (federal, state, and local) and nongovernmental entities
  • Prioritized surgical and medical indications listed for CPG development
  • State the role of opioid alternatives as first-line therapies, specify any other interventions, including nonopioid interventions, used to relieve pain


Capture.JPGGreater Generic Competition and Lower Generic Drug Prices

  • New analysis showing greater competition among generic drug makers associated with lower generic drug prices
  • With six or more competitors,  price reductions of >95% compared to brand prices before generic entry
  • FDA helping bring greater efficiency and transparency to generic drug review process to encourage competition


Capture.JPGOperation Vapor Lock seized sale of  illicit THC vaping cartridges 

FDA and DEA have seized 44 websites advertising the sale of illicit THC vaping cartridges 

  • Website advertising under various brand names with information indicating sale items would be considered a controlled substance under federal law
  • Some websites solely to fraudulently obtain payments without intending to mail product


Capture.JPGHaving Naloxone on Hand Can Save a Life During an Opioid Overdose

Naloxone is a life-saving drug that, when sprayed into the nose or injected, quickly reverses the powerful effects of opioids during an overdose

  • Expanded availability by allowing consumers to get directly from pharmacist, by putting a “standing order”
  • Need to recognize opioid overdose and use Naloxone
  • Will not harm if no opioids in system
  • Discuss Naloxone when getting opioid prescription


Capture.JPG2019-2020 Influenza season

Flu vaccine lots that have been released by FDA and are available for distribution by the manufacturers


Image credit: FDA

Drug Authorizations: OXBRYTA and ADAKVEO for sickle cell, XCOPRI for partial-onset seizures, CALQUENCE for leukemia/lymphoma, GIVLAARI for acute hepatic porphyria


OXBRYTA (voxelotor) tablets

Global Blood Therapeutics

INDICATION FOR USE: Treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.

ADDRESSING UNMET NEED: Treatment option for 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder

MECHANISM OF ACTION: Hemoglobin S (HbS) polymerization inhibitor that binds to HbS with a 1:1 stoichiometry and exhibits preferential partitioning to red blood cells (RBCs) – increasing the affinity of Hb for oxygen


  • Randomized, double-blind, placebo-controlled, multicenter trial, n=274 patients with sickle cell disease, OXBRYTA vs. placebo
  • Endpoint: Hb response rate defined as a Hb increase of >1 g/dL from baseline to Week 24
  • 51.1% (OXBRYTA) vs. 6.5% (placebo),  (p < 0.001); no outlier subgroups observed


  • Common side effects: Headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia


  •  Accelerated Approval, Fast Track designation. Orphan Drug designation
  • Accelerated approval requirements: Phase 3, randomized, doubleblind, placebo-controlled trial in pediatric patients (age 2 years to < 15 years) with Sickle Cell Disease , long-term (5 years) followup



ADAKVEO (crizanlizumab-tmca) injection


INDICATION FOR USE: Reduce the frequency of vasoocclusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease.

ADDRESSING UNMET NEED: First targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis- a painful condition

MECHANISM OF ACTION: Humanized IgG2 kappa monoclonal antibody that binds to P-selectin and blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes.


  • 52-week, randomized, multicenter, placebo-controlled, double-blind study, n=198 patients with sickle cell disease, any genotype, ADAKVEO vs placebo
  • Endpoint: Annual rate of vaso-occlusive crisis (VOC) to a healthcare visit
  • Median annual rate of 1.63 visits (ADAKVEO) vs. 2.98 visits (placebo)
  • 36%  on Adakveo did not experience VOC during the study, delayed the time of first VOC experience


  • Common side effects: Back pain, nausea, pyrexia, arthralgia
  • Need to monitor patients for infusion-related reactions,  interference with automated platelet counts or platelet clumping


  •  Priority Review and Breakthrough Therapy designation, Orphan Drug designation
  • Postapproval requirements: Further evaluation of immune mediated safety



XCOPRI® (cenobamate tablets)

SK Lifesciences

INDICATION FOR USE: Treatment of partial-onset seizures in adult patients

ADDRESSING UNMET NEED: New option to treat adults with partial-onset seizures, which is an often difficult-to-control condition that can have a significant impact on patient quality of life

MECHANISM OF ACTION: Precise mechanism unknown; demonstrated to reduce repetitive neuronal firing by inhibiting voltage-gated sodium currents


  • Two multicenter, randomized, double-blind, placebo-controlled studies, N=655 adult patients
  • Endpoint: % change from baseline in seizure frequency per 28 days in the treatment period
  • 55.6%  reduction (XCOPRI 200 mg) vs 21.5% reduction (placebo), p< 0.0001


  • Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS),  shortening of the QT interval and ventricular fibrillation – dose needs to be titrated
  • Increased risk of suicidal thoughts or behavior and other neurological adverse reactions
  • Most common side effects: Somnolence (sleepiness), dizziness, fatigue, diplopia (double vision), headaches


  • Contains cenobamate – (Controlled substance schedule to be determined after review by the Drug Enforcement Administration
  • Required pediatric assessments


CaptureCapture.JPGCALQUENCE® (acalabrutinib) capsules


INDICATION FOR USE: treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)


  • Two randomized, actively controlled trials in patients with CLL
  • Endpoint:  Progression-free survival (PFS) as assessed by independent review; significantly improved in both acalabrutinib arms, p<0.0001
  • Most common adverse reactions: Anemia, neutropenia, thrombocytopenia, headache, upper respiratory tract infection, diarrhea


  • First approved in 2017 
  • This review conducted under Project Orbis, framework for concurrent submission and review by FDA, the Australian Therapeutic Goods Administration, and Health Canada
  • FDA review used the Real-Time Oncology Review (RTOR) and Assessment Aid pilot programs, to sreamline data submission, Priority Review and Breakthrough Therapy designation


CaptureGIVLAARI (givosiran) injection

Alnylam Pharmaceuticals

INDICATION FOR USE: Treatment of adults with acute hepatic porphyria (AHP)

MECHANISM OF ACTION: Double-stranded small interfering RNA that causes degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes;  leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid
(ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP


  • Randomized, double‑blind, placebo‑controlled, multinational trial, n=94 patients with AHP, GIVLAARI vs placebo
  • Endpoint: Rate of porphyria attacks requiring hospitalizations, urgent healthcare visit, or intravenous hemin administration at home
  • 1.9 (95% CI:1.3,2.8) with GIVLAARI vs, 6.5 (95% CI:4.5, 9.3) with placebo
  • 70% (95% CI: 60%, 80%) fewer porphyria attacks with GIVLAARI


  • Most common adverse reactions: Nausea and injection site reactions
  • Warnings for anaphylactic reactions, hepatic and renal toxicities, and injection site reactions


  • Priority Review, Orphan product, Breakthrough Therapy designations
  • Postmarketing commitments: Trial in pediatric patients age greater than or equal to 12 years to less than 17 years with AHP


Image credit: Global Blood Therapeutics, Novartis, SK Lifesciences, AstraZeneca, Alnylam