🧬 This Week in Rare Diseases: FDA Leadership, Gene Therapy Safety, New Drug Approvals and more.
This week’s Rare Disease roundup delivers high-impact developments from key FDA regulatory changes and new drug approvals to gene therapy safety probes, significant clinical trial results, and multiple new orphan drug designations. Whether you’re in biotech strategy, clinical development, regulatory affairs, or advocacy—these updates keep you on the cutting edge of rare disease innovation.
🔬 Key highlights:
🚨 Vinay Prasad has stepped down as FDA CBER director amid controversy, less than three months into his term.
đź’§ The FDA approved Apellis’ EMPAVELI as the first treatment for rare kidney diseases C3G and primary IC-MPGN in patients 12 and older.
đź’Ş Gefurulimab shows positive Phase 3 results for generalized myasthenia gravis, demonstrating significant improvements in daily living activities.
⚠️ The U.S. FDA is investigating a new death linked to Sarepta’s Elevidys gene therapy, marking the fourth fatality for a Sarepta gene therapy.
🇪🇺 The EMA’s CHMP issued a negative opinion on conditional marketing authorization for Elevidys in ambulatory Duchenne muscular dystrophy patients.
đź’Š The U.S. FDA approved PTC Therapeutics’ SEPHIENCE for treating phenylketonuria (PKU) in children and adults aged one month and older.
🎗️ Sanofi’s SAR446523 earned U.S. Orphan Drug Designation for relapsed or refractory multiple myeloma.
🧬 MetrioPharm’s MP1032 received EMA Orphan Drug Designation for Duchenne muscular dystrophy, aiming to minimize corticosteroid reliance.
🩸 Kedrion Biopharma received FDA Orphan Drug Designation for COAGADEX for acquired Factor X Deficiency, a rare hematologic disorder.
Whether you’re a clinician, biotech investor, regulator, or advocate—this episode delivers the insights you need to navigate and shape the future of rare disease research and access.
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