CAMBRIDGE, Mass. — Scholar Rock, a global biopharmaceutical company focused on neuromuscular diseases, has resubmitted its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for apitegromab, a treatment for children and adults with spinal muscular atrophy (SMA).
The resubmission includes a new manufacturing facility for the drug’s final filling and packaging stages, replacing the Catalent Indiana facility that prompted the company to withdraw its previous application in August 2026 because of manufacturing compliance issues.
The alternate U.S.-based facility is the same site that supported the U.S. Food and Drug Administration’s approval of apitegromab, marketed as ISEMBYLD, on Sept. 11, 2026.
Scholar Rock expects the EMA’s Committee for Medicinal Products for Human Use (CHMP) to issue an opinion on the application during the first half of 2027.
“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,” said 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. 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.”
Apitegromab is a muscle-targeted therapy designed to improve motor function in patients already receiving treatments targeting survival motor neuron 2 (SMN2).
In the Phase 3 randomized, placebo-controlled SAPPHIRE study, patients receiving apitegromab demonstrated clinically meaningful improvements in motor function after one year, while those receiving SMN2-targeted treatment alone experienced a decline in motor function, according to the company.
Scholar Rock withdrew its original European application in August after manufacturing compliance deficiencies were identified at the Catalent Indiana fill-finish facility, which is part of Novo Nordisk.
The replacement manufacturing site produces several commercial pharmaceutical products and has undergone successful inspections by both the FDA and EMA, according to Scholar Rock.
The company said sufficient supplies of apitegromab are available at the alternate facility, with vials already at a third-party provider awaiting packaging and labeling for European commercialization if the treatment receives approval from the European Commission.
Scholar Rock specializes in developing therapies for rare and severe neuromuscular diseases using its research platform focused on myostatin biology.
