Rare Diseases
🇺🇸 FDA Page 5FDA news and analysis for pharmaceutical BD, investment, and market access · 12 articles on this page
Gain critical insights into global orphan drug development & rare disease market trends. Stay ahead in pharma R&D, investment, and regulatory strategy.
Showing 49–60 of 81 articles
FDA Grants Priority Review for IMAAVY (Nipocalimab-aahu) as First Potential Treatment for Warm Autoimmune Hemolytic Anemia
Ensartinib Phase 3 Results: Market Impact in ALK-Positive NSCLC US
FDA Approves Otarmeni: First Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program
Tivic Health Rebrands as Valion Bio, Advances Entolimod for Acute Radiation Syndrome with FDA Fast Track Status
Capricor Therapeutics HOPE-3 Phase 3 Trial Results for Deramiocel in Duchenne Muscular Dystrophy to be Presented at AAN 2026
Fierce Biotech Week 2026: What to Expect in Boston
Alterity Therapeutics ATH434 Shows Reduced Functional Decline in Multiple System Atrophy Phase 2 Trial
FDA Issues Complete Response Letter for Atara Biotherapeutics' EBVALLO Cancer Treatment, Triggering Securities Lawsuit
Veloxis Pharmaceuticals' Pegrizeprument Receives FDA Orphan Drug Designation for Heart Transplant Rejection Prevention
FDA Breakthrough Therapy Designation: Impact on Rare Disease Investment & Innovation
Pediatric Clinical Trials: FDA's Updated Requirements and Incentives
Denali Therapeutics Receives FDA Approval for Avlayah (Tividenofusp Alfa) to Treat Hunter Syndrome