Cambridge, Mass. — Scholar Rock said the FDA review of its Biologics License Application for apitegromab, an experimental treatment for spinal muscular atrophy, is continuing with a second fill-finish manufacturing facility after the company moved to remove a Catalent Indiana site from the application.
The company said the application remains on track for a potential FDA decision by its Sept. 30, 2026 Prescription Drug User Fee Act action date.
Scholar Rock said it was notified by Catalent Indiana LLC on Aug. 7 that the FDA had classified an April inspection of the facility as Official Action Indicated. The company said it is working with the FDA and, under the agency’s guidance, will remove the Catalent facility from the apitegromab application.
Scholar Rock originally included two fill-finish facilities in its March 2026 BLA submission, providing separate manufacturing options for a potential commercial launch.
The FDA is now reviewing the second U.S.-based fill-finish facility. Scholar Rock said it submitted the required data package ahead of the timeline agreed upon with the agency during a Type C meeting in March.
“We continue to be very pleased with our second fill-finish facility,” said David L. Hallal, Board Chair and Chief Executive Officer of Scholar Rock. “The data package that we agreed upon with the FDA at our Type C meeting has been submitted, the FDA review of the facility is advancing, robust supply for commercialization is ready and awaiting packaging and labeling at a third-party site, and importantly, the facility has demonstrated a strong track record of compliance, including recent successful FDA and EMA inspections.”
Scholar Rock said commercial supplies of apitegromab produced at the second facility are already available, with vials at a third-party provider awaiting packaging and labeling if the treatment receives FDA approval.
The company said the second manufacturing facility produces several commercial products and is in good standing with both the FDA and European Medicines Agency.
Apitegromab is being developed as a muscle-targeted therapy for children and adults with spinal muscular atrophy, a rare neuromuscular disease.
Scholar Rock said it is also working with the European Medicines Agency to include the second fill-finish facility in its apitegromab Marketing Authorisation Application. The company plans to provide an updated timeline for a European regulatory opinion after reaching alignment with the agency.
