Scholar Rock Drops Catalent Indiana to De-Risk FDA Review of Spinal Muscular Atrophy Candidate Apitegromab

Scholar Rock Drops Catalent Indiana to De-Risk FDA Review of Spinal Muscular Atrophy Candidate Apitegromab

Scholar Rock has executed a major strategic realignment of its global manufacturing and regulatory plans for apitegromab, its investigational muscle-strengthening monoclonal antibody. This realignment was prompted by critical regulatory and manufacturing challenges at its primary contract manufacturing site, Catalent Indiana LLC (a facility owned by Novo Nordisk).

While the company has successfully de-risked its U.S. regulatory pathway, the manufacturing issues have forced a complete withdrawal of its European marketing application.¹

The Catalent Indiana OAI Classification

On August 7, 2026, Scholar Rock received notification that the FDA classified its April 2026 inspection of the Catalent Indiana fill-finish facility as Official Action Indicated (OAI), an adverse regulatory classification signaling significant compliance deficiencies.

Under guidance from the FDA, Scholar Rock moved to immediately remove the Catalent Indiana facility from its Biologics License Application (BLA) for apitegromab in spinal muscular atrophy (SMA).

De-Risking the U.S. BLA Review (PDUFA: September 30, 2026)

Unlike many biotech programs that suffer multi-year delays from a manufacturing CRL, Scholar Rock had proactively de-risked its U.S. regulatory path. When resubmitting its BLA in March 2026, the company included two independent fill-finish facilities: Catalent Indiana and an alternate U.S.-based commercial-scale facility.

• Seamless Transition: Following the OAI classification, Scholar Rock removed Catalent Indiana, allowing the FDA to progress its review solely with the second fill-finish facility.
• Timeline Intact: The data package for the second facility was submitted ahead of schedule. The FDA review remains fully on track, with a Prescription Drug User Fee Act (PDUFA) target action date of September 30, 2026.
• Launch Readiness: Scholar Rock has confirmed that commercial vials from the second facility are already manufactured, on-site at a third-party provider, and awaiting packaging and labeling to support an immediate U.S. launch upon approval.

European Regulatory Withdrawal

While the U.S. review was saved by the dual-facility strategy, the European regulatory pathway suffered a severe setback. On August 24, 2026, Scholar Rock announced that it had withdrawn its European Marketing Authorisation Application (MAA) for apitegromab in SMA.

Because the European application relied solely on the Catalent Indiana site as its fill-finish facility, the EMA was holding its regulatory decision in anticipation of the FDA's inspection classification. Scholar Rock plans to provide updated European timelines once it aligns on a new manufacturing plan with the EMA, which will likely involve integrating the second U.S.-based fill-finish facility into a future European submission.

Pipeline Expansion: Fast Track in FSHD (September 2, 2026)

Despite the regulatory hurdles in Europe, Scholar Rock continues to advance apitegromab's clinical pipeline. On September 2, 2026, the company announced that the FDA granted Fast Track and Orphan Drug designations to apitegromab for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This milestone coincided with the commencement of participant dosing in the Phase 2 FORGE trial, evaluating the muscle-selective anti-myostatin antibody's potential to improve motor function in patients living with FSHD.