Biotech firm Scholar Rock Holdings said it removed Catalent Indiana LLC as a fill-finish facility from its U.S. biologics license application for apitegromab, an experimental treatment for spinal muscular atrophy.
The company stated the move follows the U.S. Food and Drug Administration’s August 7 classification of Catalent Indiana’s April 2026 inspection as Official Action Indicated, indicating significant compliance concerns. Scholar Rock is now relying on an alternate U.S.-based facility for commercial production, which has passed recent FDA and European Medicines Agency inspections and is prepared to supply vials upon approval.
Scholar Rock also withdrew its marketing authorization application for apitegromab with the EMA’s Committee for Medicinal Products for Human Use following the FDA’s inspection outcome. The company plans to resubmit the application using the alternate facility. A decision from the FDA on the BLA is expected by September 30 under the Prescription Drug User Fee Act timeline.
The drug, a monoclonal antibody designed to inhibit myostatin activation in skeletal muscle, holds 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. Scholar Rock noted that apitegromab has not yet been approved by any regulatory agency.
Japan’s Pharmaceuticals and Medical Devices Agency has indicated no additional clinical studies are required for a planned regulatory submission, with a target submission by year-end 2026.
