Scholar Rock Resubmits Apitegromab Marketing Authorisation Application (MAA) to European Medicines Agency (EMA) for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
Scholar Rock resubmitted the Marketing Authorisation Application for apitegromab to the EMA for treatment of children and adults with spinal muscular atrophy. The resubmission replaces the Catalent Indiana fill-finish facility, which had GMP compliance issues, with an alternate U.S.-based site that supported the September 2026 FDA approval of ISEMBYLD; a CHMP opinion is anticipated in H1 2027.
Key figures
- Drug Name
- apitegromab (ISEMBYLD, apitegromab-mstn)
- Phase Of Trial
- Phase 3
- Chmp opinion timing
- H1 2027
- Planned country expansion
- up to 50 countries
- Target patient population
- more than 35,000 SMA patients globally receiving an SMN-targeted therapy
AI analysis
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