Scholar Rock Drops Troubled Catalent Facility from Apitegromab BLA While Progressing Toward September 30 PDUFA Date

Scholar Rock Drops Troubled Catalent Facility from Apitegromab BLA While Progressing Toward September 30 PDUFA Date

Scholar Rock (NASDAQ: SRRK) has navigated a significant regulatory hurdle for its investigational muscle-targeted monoclonal antibody, apitegromab, which is under review for the treatment of children and adults with spinal muscular atrophy (SMA). On August 7, 2026, the company announced that it is removing Novo Nordisk's Catalent Indiana fill-finish facility from its Biologics License Application (BLA) following a negative regulatory inspection of the site. Despite this change, the FDA's review remains on track to meet its September 30, 2026 Prescription Drug User Fee Act (PDUFA) target action date.

Dropping the Catalent Indiana Facility

Scholar Rock's March 2026 BLA resubmission for apitegromab originally included two independent fill-finish facilities to provide parallel pathways to approval.¹ However, on August 7, 2026, Catalent Indiana LLC notified Scholar Rock that the FDA had classified its April 2026 site inspection as Official Action Indicated (OAI), a serious regulatory status indicating that significant objectionable conditions were found.

Under FDA guidance, Scholar Rock has formally removed the Catalent Indiana facility from the BLA. The review will now progress solely with the second, alternative U.S.-based fill-finish facility, which is currently in good standing with both the FDA and EMA. Robust commercial supply is already produced and awaiting final packaging and labeling at a third-party site upon approval.

In Europe, the Catalent inspection delay prompted Scholar Rock to temporarily withdraw its Marketing Authorisation Application (MAA) from the European Medicines Agency (EMA), with plans to refile once the second fill-finish facility is officially incorporated into the application.

Pipeline Expansion: FDA Fast Track and Phase 2 FORGE Trial in FSHD

On September 2, 2026, Scholar Rock announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for a second indication: the treatment of facioscapulohumeral muscular dystrophy (FSHD), a rare genetic muscle-wasting disorder. Simultaneously, the company announced that participant dosing has officially commenced in the Phase 2 FORGE trial, a randomized, double-blind, placebo-controlled multi-center study evaluating the efficacy, safety, and pharmacodynamics of apitegromab monotherapy in adults with genetically confirmed FSHD.