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Vitrakvi (larotrectinib) capsules and oral solution

 Loxo Oncology

INDICATION: Treatment of adult and pediatric patients with solid tumors that:

  • have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation
  • are metastatic or where surgical resection is likely to result in severe morbidity
  • have no satisfactory alternative treatments or that have progressed following treatment

ADDRESSING UNMET NEED:

  • Treatment based on a common biomarker across different types of tumors rather than location of tumor
  • Treatment for cancers based on tumor genetics rather than site of origin
  • New paradigm in the development of cancer drugs that are “tissue agnostic”

MECHANISM OF ACTION: Inhibitor of the tropomyosin receptor kinases (TRK), TRKA, TRKB, TRKC; TRK fusion proteins can act as oncogenic driver, promoting cell proliferation and survival in tumor cell lines

EFFICACY:

  • Three multicenter, open-label, single-arm clinical trials, n= 55 pediatric and adult patients with solid tumors with NTRK gene fusion with no satisfactory alternative treatments
  • Responding tumor types: Soft tissue sarcoma, salivary gland cancer, infantile fibrosarcoma, thyroid cancer and lung cancer
  • Overall response rate across different types of solid tumors: 75%
  • Response duration: 73% lasting at least six months, 39% lasting a year or more

SAFETY:

  • Common side effects: Fatigue, nausea, cough, constipation, diarrhea, dizziness, vomiting, increased AST and ALT enzyme blood levels in the liver
  • May cause harm to a developing fetus or newborn baby

REGULATORY PATHWAY: NDA

  • Accelerated approval based on specific genetic feature (biomarker); continued approval contingent upon verification and description of clinical benefit in confirmatory trials
  • Accelerated approval requirement: Verify and describe clinical benefit in adult and pediatric patients
  •  Priority Review, Breakthrough Therapy designation, Orphan Drug designation
  • Rare pediatric disease priority review voucher  denied

LABEL


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FIRDAPSE (amifampridine) tablets

 Catalyst Pharmaceuticals

INDICATION: Treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults

ADDRESSING UNMET NEED:

  • First treatment for LEMS
  • Rare autoimmune disorder affecting connection between nerves and muscles
  • Causes weakness and other symptoms in affected patients

MECHANISM OF ACTION: Not fully elucidated; broad spectrum potassium channel blocker.

EFFICACY:

  • Two clinical trials, n=64 adult patients, Firdapse vs. placebo
  • Endpoint: Quantitative Myasthenia Gravis score (a 13-item physician-rated categorical scale assessing muscle weakness) and Subject Global Impression (a seven-point scale on which patients rated their overall impression of the effects of the study treatment on their physical well-being)
  • Patients receiving Firdapse experienced a greater benefit vs. placebo

SAFETY:

  • Most common side effects: Burning or prickling sensation (paresthesia), upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension and muscle spasms
  • Seizures and  hypersensitivity reactions such as rash, hives, itching, fever, swelling or trouble breathing

REGULATORY PATHWAY: NDA

  • Priority Review, Breakthrough Therapy designations, Orphan Drug designation

LABEL


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XOSPATA (gilteritinib) tablets

Astellas

INDICATION: Treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test

ADDRESSING UNMET NEED:

  • ~ 25-30% AML patients have FLT3 gene mutation; associated with aggressive form of disease and higher risk of relapse
  • First drug to be used alone in treating patients with AML having FLT3 mutation

MECHANISM OF ACTION: Inhibits multiple receptor tyrosine kinases, including FLT3

EFFICACY:

  •  Clinical trial, n=138 patients with relapsed or refractory AML with confirmed FLT3 mutation
  • Complete remission (no evidence of disease and full recovery of blood counts) or complete remission with partial hematologic recovery (no evidence of disease and partial recovery of blood counts) : 21%
  • Of the 106 patients requiring RBC or platelet transfusions; 31% transfusion-free for at least 56 days

SAFETY:

  • Common side effects: Muscle and joint pain (myalgia/arthralgia), fatigue and elevated liver enzymes (liver transaminase)
  • Required monitoring for posterior reversible encephalopathy syndrome, prolonged QT interval, pancreatitis (inflammation in the pancreas)
  • May cause harm to a developing fetus or newborn baby.

REGULATORY PATHWAY: NDA

  •  Fast Track, Priority Review designation, Orphan Drug designation
  • Postmarketing requirements: Clinical assessment of long-term safety and risks or rare adverse events (differentiation symdrome)

LABEL


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reSET-O App

Pear Therapeutics

INDICATION FOR USE:  Prescription cognitive behavioral therapy intended to be used in addition to outpatient treatment under the care of a health care professional, in conjunction with treatment that includes buprenorphine and contingency management

ADDRESSING UNMET NEED:

  • As part of FDA efforts to address the misuse and abuse of opioids
  • New tool to help more people with opioid use disorder successfully treat their addiction

DESCRIPTION:

  • App can be downloaded directly to a patient’s mobile device
  • Used while participating in an outpatient Opioid Use Disorder (OUD) treatment program
  • For training, monitoring and reminder tool for health care providers and patients
  • Compliance reward system– such as earning special icons on a prize wheel

EFFECTIVENESS & SAFETY:

  •  Multi-site, unblinded, controlled 12-week clinical trial, n=170 patients, with or without reSET-O
  • Supervised administration of buprenorphine and urine screens; contingency management system to reward negative urine tests
  • Illicit drug use, abstinence improvement : No decrease  with reSET-O vs. buprenorphine treatment and contingency management alone
  • Retention in treatment program for 12 weeks: 82.4%  with reSET-O vs. 68.4% without reSET-O
  • Adverse events: Typical of patients with OUD – cardiovascular disease, gastrointestinal diseases, HIV, Hepatitis C, nutritional diseases, risk of overdose, depression, mania, suicidal behavior and suicidal ideation and attempts

REGULATORY PATHWAY: 510(k)

  • Predicate device, reSET App- classified via De Novo pathway in 2017
  • Regulation Number : 882.5801
  • Classification : II
  • Classification Name: Computerized Behavioral Therapy Device For Psychiatric Disorder
  • Product Code: PWE

Regulation


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Truxima (rituximab-abbs)

Celltrion Inc.

INDICATION: Non–Hodgkin’s Lymphoma (NHL). Treatment of adult patients with:

  • Relapsed or refractory, low-grade or follicular, CD20-positive, B-cell NHL as a single agent
  • Previously untreated follicular, CD20-positive, B-cell NHL in combination with first line chemotherapy and, in patients achieving a complete or partial response to a rituximab product in combination with chemotherapy, as single-agent maintenance therapy
  • Non-progressing (including stable disease), low-grade, CD20-positive, B-cell NHL as a single agent after first-line cyclophosphamide, vincristine, and prednisone (CVP) chemotherapy

ADDRESSING UNMET NEED & REGULATORY PATHWAY:

  • First biosimilar to Rituxan (rituximab, Genentech Inc.)  
  • Approval based on a review of extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, other clinical data
  • Demonstrated that Truxima is biosimilar to Rituxan
  • Not as an interchangeable product.

LABEL


Image credit: Loxo, Catalyst, Astellas, Pear, Celltrion

 

 

 

 

 

 

 

 

 

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