Scholar Rock Resubmits Apitegromab Marketing Authorisation Application (MAA) to European Medicines Agency (EMA) for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
Business Wire•08/10/2026•08:00 ET
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Key Highlights
- ➤Apitegromab MAA resubmitted to EMA with an alternate fill-finish facility
- ➤Alternate facility supported September 2026 FDA approval of ISEMBYLD
- ➤CHMP opinion anticipated in the first half of 2027
- ➤Previous MAA withdrawn over Catalent Indiana facility’s lack of GMP compliance
- ➤Apitegromab targets more than 35,000 globally treated SMA patients
Expert Statements
David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock
“With the launch of ISEMBYLD now underway in the U.S., we are working with urgency to bring this first-ever muscle-targeted therapy to children and adults living with SMA in Europe.”
David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock
“Consistent with our ongoing dialogue with EMA, we are pleased with the expeditious resubmission of our apitegromab MAA.”
David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock
“This marks a decisive step forward in advancing our mission to bring apitegromab to the more than 35,000 SMA patients globally who are receiving an SMN-targeted therapy.”
Scholar Rock Resubmits Apitegromab Marketing Authorisation Application (MAA) to European Medicines Agency (EMA) for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
* Apitegromab MAA resubmission includes alternate fill-finish facility in place of Catalent Indiana LLC (part of Novo Nordisk), in alignment with guidance from EMA
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