CAMBRIDGE, Mass., August 7, 2026
Scholar Rock announced that the U.S. Food and Drug Administration (FDA) review of its Biologics License Application (BLA) for apitegromab for children and adults with spinal muscular atrophy (SMA) is progressing with the company’s second fill-finish facility. The company said the BLA remains on track for a potential FDA decision by the September 30, 2026 PDUFA action date, following the removal of Catalent Indiana LLC from the application after the FDA classified its April 2026 facility inspection as Official Action Indicated (OAI).
FDA Review Continues with Second Fill-Finish Facility
Scholar Rock said it is working closely with the FDA to remove Catalent Indiana, a facility operated by Novo Nordisk, from the apitegromab BLA. The FDA review will now proceed solely with the company’s second fill-finish facility. Scholar Rock stated that the second facility is a U.S.-based manufacturing site producing multiple commercial products and remains in good standing with both the FDA and European Medicines Agency (EMA).
Second Facility Data Package Submitted Ahead of Schedule
At a March 2026 Type C meeting, Scholar Rock and the FDA agreed on the data package required to support regulatory review of the second fill-finish facility. The company has now submitted that package ahead of the agreed timeline, and said FDA review is progressing. Scholar Rock also reported that a robust supply of commercial apitegromab vials from the second facility is already available at a third-party site and awaiting packaging and labeling, potentially supporting a U.S. launch following regulatory approval.
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Apitegromab Positioned for Potential SMA Launch
Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding to the pro- and latent forms of myostatin in skeletal muscle. The therapy is being developed as a muscle-targeted treatment for SMA and has demonstrated clinical success in a pivotal Phase 3 trial. Scholar Rock said the FDA has granted apitegromab Fast Track, Orphan Drug, and Rare Pediatric Disease designations, while the EMA has granted PRIME and Orphan Medicinal Product designations.
European Regulatory Review Also Being Updated
Scholar Rock is also engaging with the EMA regarding the inclusion of its second fill-finish facility in the company’s Marketing Authorisation Application (MAA) for apitegromab in Europe. The company expects to provide updated timelines for a Committee for Medicinal Products for Human Use (CHMP) opinion once alignment with the EMA has been reached.
Source: Scholar Rock, release
