Rare Diseases
🇪🇺 EMA Page 2EMA 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 13–24 of 31 articles
Norgine Receives European Commission Approval for XOLREMDI (Mavorixafor) as First WHIM Syndrome Treatment
EU HTA Regulation Impact: Effects on Rare Disease Drug Approvals & Market Access
European Biomarker-Driven Trials: EMA Regulatory Framework & Success Rates
Solid Biosciences Receives European Orphan Drug Designation for SGT-003 Duchenne Muscular Dystrophy Treatment
EU Pharmaceutical Legislation Reform: Impact on Orphan Drug Development & Market Exclusivity
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
EMA Recommends Approval of Plozasiran (Redemplo) for Rare Triglyceride Disorder FCS
CLINUVEL Receives EMA Approval for SCENESSE Phase III Vitiligo Trial Starting 2026
Pembrolizumab Plus Lenvatinib Shows Promise for Rare Clear Cell Gynecological Cancers in Phase 2 Trial
Polycythemia Vera Market Transformation: 9 Companies Including Protagonist Therapeutics with Rusfertide to Enter by 2036