Scholar Rock Resubmits SMA Drug Application to EMA After Manufacturing Switch
Event summary
- Scholar Rock resubmitted its Marketing Authorisation Application (MAA) for apitegromab to the EMA for treating SMA, replacing the Catalent Indiana fill-finish facility with an alternate site.
- The resubmission follows the withdrawal of the initial MAA in August 2026 due to GMP compliance issues at Catalent Indiana.
- Scholar Rock anticipates a CHMP opinion in the first half of 2027, with robust supply available from the alternate U.S.-based facility.
- Apitegromab, marketed as ISEMBYLD in the U.S., is the first muscle-targeted therapy for SMA, approved by the FDA in September 2026.
The big picture
Scholar Rock’s resubmission of the apitegromab MAA to the EMA marks a critical step in expanding access to the first muscle-targeted therapy for SMA beyond the U.S. The switch to an alternate manufacturing facility underscores the challenges of regulatory compliance in biopharmaceutical production. Success in Europe would position Scholar Rock as a key player in the growing market for SMA treatments, which includes over 35,000 patients currently receiving SMN-targeted therapies.
What we're watching
- Regulatory Timeline
- Whether the EMA will meet Scholar Rock’s anticipated timeline for a CHMP opinion in the first half of 2027.
- Manufacturing Compliance
- How the alternate fill-finish facility will perform under EMA scrutiny following the previous GMP compliance issues.
- Market Expansion
- The pace at which Scholar Rock can secure approvals and commercialize apitegromab in up to 50 countries globally.