Rare Diseases News
🇪🇺 EMAEMA breaking news and regulatory updates for BD, investors, and analysts
Showing 1–12 of 21 articles
CHMP Negative Opinion on Acadia's Rett Syndrome Drug: Implications for Investors and Pharma Teams
X4 Pharmaceuticals Secures Positive EMA CHMP Opinion for Mavorixafor in WHIM Syndrome
Takeda's TAK-881 Meets Primary Endpoint in Phase 2/3 Trial for Primary Immunodeficiency Disease
BioCryst Secures $70M Upfront in European Licensing Deal for Navenibart Hereditary Angioedema Treatment
Stoke Therapeutics Zorevunersen Shows Promise for Dravet Syndrome Treatment Ahead of Q1 2026 Results
HUTCHMED's Sovleplenib Receives Priority Review and Breakthrough Designation in China for Rare Blood Disorder
Ray Therapeutics RTx-015 Receives EMA PRIME Designation for Retinitis Pigmentosa Treatment
Norgine Receives European Commission Approval for XOLREMDI (Mavorixafor) as First WHIM Syndrome Treatment
Solid Biosciences Receives European Orphan Drug Designation for SGT-003 Duchenne Muscular Dystrophy Treatment
Pharma 2026: Key Takeaways from EU Biopharma & Medtech
Reuters Pharma EU: Funding & Dealmaking Key Takeaways
EMA Issues Positive Opinion for Itvisma (Onasemnogene Abeparvovec) Gene Therapy for Spinal Muscular Atrophy