CAMBRIDGE, Mass. — Scholar Rock said Friday it has removed Catalent Indiana LLC, part of Novo Nordisk, as a commercial fill-finish facility from its U.S. Biologics License Application for apitegromab, its investigational treatment for spinal muscular atrophy.
The company said the change was made under guidance from the U.S. Food and Drug Administration and that the agency’s review of the application is continuing with an alternate fill-finish facility. Scholar Rock continues to expect an FDA decision by the drug’s Sept. 30, 2026, Prescription Drug User Fee Act date.
Scholar Rock said it is prepared to launch apitegromab in the United States immediately if approved and has a supply of commercial vials produced at the alternate facility.
The company also withdrew its Marketing Authorisation Application in Europe after the FDA classified an April 2026 inspection of the Catalent Indiana facility as Official Action Indicated. The withdrawal was completed through a written procedure with the European Medicines Agency’s Committee for Medicinal Products for Human Use that concluded Aug. 20.
Scholar Rock plans to remove Catalent Indiana from the European application and resubmit it using the alternate fill-finish facility, which it said should provide the fastest path toward a CHMP opinion.
In Japan, the company said it is on track to submit a Japanese New Drug Application for apitegromab by the end of 2026 after reaching agreement with the Pharmaceuticals and Medical Devices Agency that no additional clinical studies involving Japanese patients will be required.
“We are very pleased with the continued constructive and collaborative engagement we have with regulators both in the U.S. and Europe and look forward to commencing our U.S. launch in Q3 pending FDA approval,” said David L. Hallal, Board Chair and Chief Executive Officer of Scholar Rock.
Hallal said the company also plans to pursue apitegromab access in as many as 50 countries and described the agreement with Japanese regulators as another important step toward that goal.
Clinical studies involving Japanese patients are typically required for a JNDA. However, Scholar Rock said the PMDA determined that apitegromab could qualify under recent guidelines allowing some orphan and rare disease drugs to be submitted without domestic clinical trial data.
Those criteria include the successful completion of a pivotal trial outside Japan, difficulty recruiting enough patients because of a small disease population and sufficient scientific and clinical evidence suggesting a favorable benefit-risk profile for Japanese patients.
Scholar Rock said its alternate fill-finish facility is based in the United States and manufactures multiple commercial products. The site has also undergone recent successful FDA and EMA inspections.
Apitegromab is a fully human monoclonal antibody designed to inhibit activation of myostatin by selectively binding its pro- and latent forms in skeletal muscle. Scholar Rock is developing the therapy as a muscle-targeted treatment for SMA.
The drug has received Fast Track, Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as Priority Medicines and Orphan Medicinal Product designations from the EMA.
Apitegromab has not been approved by the FDA or any other regulatory agency.
