Massachusetts-based Scholar Rock (Nasdaq: SRRK) announced that the FDA review of its biologics license application (BLA) for apitegromab, a selective anti-myostatin monoclonal antibody for spinal muscular atrophy (SMA), will proceed with a second fill-finish facility supporting the application after Catalent Indiana LLC received an Official Action Indicated (OAI) classification following an April 2026 FDA site inspection. The PDUFA action date of September 30, 2026 remains in place, the company said.
Scholar Rock's March 2026 BLA submission named two fill-finish facilities — Catalent Indiana (part of Novo Nordisk) and a second, undisclosed US-based facility — providing independent manufacturing pathways. Following the OAI classification, Scholar Rock said it will remove Catalent Indiana from the BLA under FDA guidance, with review continuing exclusively through the second facility, which the company said holds good standing with both the FDA and the European Medicines Agency (EMA).
The data package for FDA review of the second facility, agreed upon at a March 2026 Type C meeting, was submitted ahead of the agreed timeline, the company reported. Commercial supply vials from that facility are on-site at a third-party provider awaiting packaging and labeling. Scholar Rock also said it is engaging with the EMA on steps to include the second facility in the apitegromab Marketing Authorisation Application (MAA), with updated Committee for Medicinal Products for Human Use (CHMP) timelines to follow.
Apitegromab is a fully human IgG4 monoclonal antibody designed to selectively inhibit activation of myostatin, a negative regulator of skeletal muscle growth. The approach is intended to complement existing SMN-directed therapies by targeting muscle atrophy independently of SMN protein restoration.