Rare Diseases Update – December 4, 2025: SMA gene therapy OK’d, Regeneron–Tessera AATD pact and More

Описание: From the United States to the European Union and Canada, this concise roundup spotlights pivotal regulatory milestones, policy shifts, and market signals shaping the rare pipeline over the past two weeks.

🔬 Key highlights:

🧠 Novartis/Itvisma: FDA approved intrathecal onasemnogene abeparvovec for SMA in patients 2+ years, including adults

🍁 CORD/Canada: Midpoint report says Phase 1 funded at least 12 drugs across nine conditions, urges more screening and RWD

💊 EMD Serono: FDA Fast Track for oral cladribine capsules in generalized myasthenia gravis, Phase 3 ongoing

🌐 EU policy op-ed: Calls for AI-enabled, digitally enhanced rare disease care aligned with the European Health Data Space

🧬 Solid Biosciences: FDA Rare Pediatric Disease and Fast Track for SGT-212 in Friedreich’s ataxia, Phase 1b screening

🧒 Latus Bio: FDA cleared IND for LTS-101 in CLN2, with Fast Track, Orphan Drug, Rare Pediatric Disease

🧪 Kedrion: EMA granted Orphan Drug Designation for plasma-derived therapy in congenital aceruloplasminemia

🤝 Regeneron–Tessera: $150M upfront and equity for TSRA-196 in AATD, plus up to $125M milestones, 50–50 cost and profit split

Chapters:
0:00 Introduction
0:08 Novartis/Itvisma: FDA approved intrathecal onasemnogene abeparvovec for SMA in patients 2+ years, including adults
0:44 CORD/Canada: Midpoint report says Phase 1 funded at least 12 drugs across nine conditions, urges more screening and RWD
1:08 EMD Serono: FDA Fast Track for oral cladribine capsules in generalized myasthenia gravis, Phase 3 ongoing
1:34 EU policy op-ed: Calls for AI-enabled, digitally enhanced rare disease care aligned with the European Health Data Space
1:50 Solid Biosciences: FDA Rare Pediatric Disease and Fast Track for SGT-212 in Friedreich’s ataxia, Phase 1b screening
2:17 Latus Bio: FDA cleared IND for LTS-101 in CLN2, with Fast Track, Orphan Drug, Rare Pediatric Disease
2:40 Kedrion: EMA granted Orphan Drug Designation for plasma-derived therapy in congenital aceruloplasminemia
3:01 Regeneron–Tessera: $150M upfront and equity for TSRA-196 in AATD, plus up to $125M milestones, 50–50 cost and profit split
3:33 How to reach us

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