🧬 This Week in Rare Diseases: Europe’s R&D at Risk, AI Drug Discovery, New FDA Approvals, and More
This week’s Rare Diseases roundup brings you high-impact updates spanning policy threats to European innovation, groundbreaking AI drug development partnerships, pivotal FDA approvals, and major clinical trial milestones. Whether you work in biotech strategy, clinical development, regulatory affairs, or patient advocacy—these insights will keep you at the forefront of rare disease innovation.
🔬 Key highlights:
🌍 Europe’s rare disease innovation progress at risk as Chiesi’s Enrico Piccinini warns reduced EU incentives could push R&D overseas, calling for stronger IP protections and better public–private collaboration.
🎥 Alexion launches Rare Connections in gMG, an awareness film featuring patient stories to educate on the fluctuating, often invisible symptoms of generalized myasthenia gravis.
🇿🇦 AstraZeneca enters rare disease care in South Africa with new treatments for NF1, PNH, aHUS, and gMG, focusing on accelerating access and improving diagnosis in children.
🤖 Dr. Noah Biotech and Thermo Fisher explore AI integration with organoid-based testing to speed rare disease drug development, building on Dr. Noah’s FDA-designated ALS therapy.
💉 FDA approves Novo Nordisk’s Alhemo for hemophilia A and B without inhibitors, cutting bleeding rates by up to eighty-six percent in Phase 3 trials.
🧪 SGX945 shows Phase 2 success in Behçet’s Disease with sustained ulcer improvement after treatment and no major side effects, with plans for a self-administered version.
🩺 Arcus Biosciences’ quemliclustat gains orphan drug status for pancreatic cancer, showing median overall survival of 15.7 months in Phase 1.
🧬 Vivace Therapeutics earns orphan designation for VT3989 in mesothelioma, the first TEAD autopalmitoylation inhibitor with strong Phase 1 efficacy and safety data.
🧩 Gene-editing therapy PBGENE-DMD secures FDA orphan and pediatric rare disease designation for Duchenne muscular dystrophy, aiming to restore dystrophin production.
🇨🇳 Sanofi acquires Chinese rights to Arrowhead’s plozasiran for rare lipid disorders in a $130M upfront deal, with Phase 3 data showing triglyceride reductions of eighty percent.
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