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BOSE HEARING AID

Bose Corporation

INDICATION FOR USE: To amplify sounds for individuals 18 years or older with perceived mild to moderate hearing impairment (hearing loss)

ADDRESSING UNMET NEED: 

  •  37.5 million adults aged 18 and over report having some trouble hearing without a hearing aid
  • First hearing aid authorized for marketing by the FDA that enables users to fit, program and control the hearing aid on their own, without assistance from a health care provider

DEVICE DESCRIPTION:

  • User-fitted wireless air conduction hearing aid
  • Captures sound vibrations through one or more microphones
  • Signal is processed, amplified, and played back through an earphone placed in the ear canal
  • Patients can adjust aid through mobile application on phone
  • Enables users to fit the hearing aid settings themselves, in real-time and in real-world environments without the assistance of a health care professional

EFFECTIVENESS & SAFETY:

  • Clinical data from 125 patients
  • Self-fitting of the Bose Hearing Aid comparable to those with professional fitting
    • Amount of amplification selected, speech in noise testing and overall benefit
  • Patients preferred those hearing aid settings over professionally-selected settings
  • Labeling to inform consumer when to consult a hearing health care professional

REGULATORY PATHWAY: De Novo request


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JIVI [antihemophilic factor (recombinant), PEGylated-aucl]

Bayer Healthcare

INDICATION FOR USE: For use in previously treated adults and adolescents (12 years of age and older) with hemophilia A (congenital Factor VIII deficiency) for: on-demand treatment and control of bleeding episodes; perioperative management of bleeding; and routine prophylaxis to reduce the frequency of bleeding episodes.

ADDRESSING UNMET NEED:

  • Option for : On-demand treatment and control of bleeding episodes; Perioperative management of bleeding;  Routine prophylaxis to reduce the frequency of bleeding episodes.

MECHANISM OF ACTION: Site-specifically PEGylated recombinant antihemophilic factor [see Description (11)], temporarily replaces the missing coagulation Factor VIII. The site-specific PEGylation in the A3 domain reduces binding to the physiological Factor VIII clearance receptors resulting in an extended half-life and increased AUC

BENEFIT/RISK:

Benefits

  • On-demand JIVI is effective for treatment of and prevention of spontaneous or traumatic bleeding in patients with Hemophilia A
  • JIVI is effective in the perioperative setting for reduction of bleeding during surgery
  • JIVI is effective in patients over years of age

Risks

  • Hypersensitivity reactions and development of anti-PEG antibodies which resulted in loss of efficacy in patients <12 years of age
  • Risk of development of inhibitory antibodies is considered an expected adverse event

REGULATORY PATHWAY: BLA

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LIBTAYO (cemiplimab-rwlc) injection

Regeneron Pharmaceuticals

INDICATION:  Treatment of patients with metastatic cutaneous squamous cell
carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation

ADDRESSING UNMET NEED: 

  • First  approval of a drug specifically for advanced CSCC
  • CSCC is the second most common human cancer in the United States with an estimated annual incidence of approximately 700,000 cases
  • Immune checkpoint inhibitors targeting the the PD-1 / PD-L1 pathway for treating a variety of tumors, from bladder to head and neck cancer, and now advanced CSCC

EFFICACY:

  • Two open label clinical trials, n=108 patients (75 with metastatic disease and 33 with locally-advanced disease)
  • Primary endpoint: Objective response rate, or the percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s) after treatment
  • 47.2% patients had tumors shrink or disappear

SAFETY:

  • Severe and Fatal Immune-Mediated Adverse Reactions
  • Infusion-Related Reactions, Embryo-Fetal Toxicity
  • Common side effects: Fatigue, rash and diarrhea
  • Must be dispensed with a patient Medication Guide

REGULATORY PATHWAY: BLA

  • Breakthrough Therapy and Priority Review designations

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VIZIMPRO (dacomitinib) tablets

Pfizer

INDICATION FOR USE:  First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved test

MECHANISM OF ACTION:  Irreversible inhibitor of the kinase activity of the human EGFR family (EGFR/HER1, HER2, and HER4) and certain EGFR activating mutations (exon 19 deletion or the exon 21 L858R substitution mutation)

EFFICACY:

  • Randomized, multicenter, open-label, active controlled trialm n= 452 patients with unresectable, metastatic NSCLC,  dacomitinib vs gefitinib
  • Significant improvement in progression-free survival; no improvement in overall response rate or overall survival were demonstrated
  • Median progression-free survival; 14.7 vs. 9.2 months p<0.0001

SAFETY:

  • Warnings & Precautions: Interstitial Lung Disease (ILD), Diarrhea, Dermatologic Adverse Reactions, Embryo-Fetal Toxicity

REGULATORY PATHWAY: NDA

  • Priority Review and Orphan Drug Designation
  • Postmarketing study:  Pharmacokinetic trial to determine an appropriate dose of dacomitinib to minimize toxicity in patients with severe hepatic impairment

LABEL


Image credit: Bose, Bayer, Regeneron, Pfizer

 

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