MedPath

Tagged News

FDA Approves First Direct-to-Consumer Pharmacogenetic Test from 23andMe

Drug Approval
  • The FDA approved 23andMe's Personal Genome Service Pharmacogenetic Reports, marking the first direct-to-consumer genetic test to help patients understand how their genetics may affect responses to over 50 common prescription and over-the-counter drugs.
  • The test analyzes 33 genetic variants associated with drug metabolism, including responses to blood thinners like clopidogrel and mental health treatments, but comes with strict limitations prohibiting its use for treatment decisions or medical advice.
  • Medical experts express caution about the clinical utility of pharmacogenetic testing for general consumers, noting that while genetic variants affect drug metabolism, the clinical interpretation and prescription implications remain unclear for most patients.

Novartis's Kymriah: Breakthrough CAR-T Therapy Priced at $475,000 Sparks Value Debate

CAR-TDrug ApprovalOncology
  • Novartis's Kymriah, the first FDA-approved CAR-T cell therapy for pediatric acute lymphoblastic leukemia, demonstrates an impressive 83% remission rate in patients who failed traditional treatments.
  • The therapy's $475,000 price tag has ignited debate among healthcare experts, with some defending it as comparable to bone marrow transplants while others argue it's excessive despite its breakthrough status.
  • Novartis has implemented innovative value-based pricing models, including outcome-based contracts where payment is contingent upon patient response and indication-specific pricing for future approvals.

FDA Approves Two New Ustekinumab Biosimilars, Bringing Total US Biosimilar Count to 65

Monoclonal AntibodyDrug Approval
  • The FDA has approved Biocon's Yesintek (ustekinumab-kfce) and Celltrion's Steqeyma (ustekinumab-stba) in November and December 2024, expanding treatment options for autoimmune conditions including Crohn's disease and psoriasis.
  • Of the 65 FDA-approved biosimilars, 14 have received interchangeable designation, allowing pharmacists to substitute them for reference products without prescriber intervention, enhancing patient access to more affordable treatments.
  • The biosimilar landscape continues to evolve with products spanning five major classes: insulin, granulocyte colony-stimulating factors, monoclonal antibodies, TNF-alpha inhibitors, and VEGF inhibitors.

FDA Approves Sanofi's Multaq for Atrial Fibrillation Patients, Marking First New Treatment in Two Decades

Drug Approval
  • The FDA has approved Sanofi's Multaq (dronedarone) to reduce cardiovascular hospitalization risk in patients with atrial fibrillation or atrial flutter, representing the first new treatment option in this therapeutic area in twenty years.
  • The approval is based on five clinical studies involving 6,300 patients, with the landmark ATHENA trial demonstrating a 24% reduction in cardiovascular hospitalization or death from any cause compared to placebo.
  • Multaq showed significant clinical benefits including a 28% reduction in total hospital days and 35% decrease in heart-related hospitalization time in atrial fibrillation patients.
  • The drug is contraindicated in patients with severe heart failure, following results from the ANDROMEDA trial that showed increased mortality risk in this population.

FDA Approves Pembrolizumab for Advanced Esophageal Squamous Cell Cancer with PD-L1 Expression

Drug Approval
  • The FDA approved pembrolizumab for patients with recurrent, locally advanced, or metastatic esophageal squamous cell carcinoma whose tumors express PD-L1 with a combined positive score of 10 or higher.
  • Clinical trials KEYNOTE-181 and KEYNOTE-180 demonstrated significant survival benefits, with median overall survival of 10.3 months versus 6.7 months for control treatment.
  • The approval includes a companion diagnostic test to identify eligible patients based on PD-L1 expression levels using the PD-L1 IHC 22C3 pharmDx kit.
  • The recommended dosing regimen is pembrolizumab 200 mg administered intravenously every 3 weeks until disease progression or unacceptable toxicity.

Study of Pembrolizumab (MK-3475) in Previously-Treated Participants With Advanced Carcinoma of the Esophagus or Esophagogastric Junction (MK-3475-180/KEYNOTE-180)

NCT02559687CompletedPhase 2
Merck Sharp & Dohme LLC
Posted 12/2/2015

Study of Pembrolizumab (MK-3475) Versus Investigator's Choice Standard Therapy for Participants With Advanced Esophageal/ Esophagogastric Junction Carcinoma That Progressed After First-Line Therapy (MK-3475-181/KEYNOTE-181)

NCT02564263CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 12/1/2015

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.