Regulatory & Policy

Scholar Rock withdraws EU apitegromab filing over manufacturing site issue

Cambridge, Massachusetts-based Scholar Rock (Nasdaq: SRRK) announced a series of regulatory updates on August 24, 2026, confirming that its Biologics...

Scholar Rock withdraws EU apitegromab filing over manufacturing site issue

Scholar Rock (Nasdaq: SRRK) said August 24 it has withdrawn the European marketing application for spinal muscular atrophy drug apitegromab after a manufacturing site received an adverse US FDA inspection classification, while its US review remains on track for a September 30, 2026 PDUFA date.

The Massachusetts biotech removed Catalent Indiana LLC, now part of Novo Nordisk, from its US Biologics License Application under US FDA guidance and will rely solely on an alternate US fill-finish facility. Catalent Indiana received an Official Action Indicated classification following an April 2026 site inspection.

Manufacturing issues at the same facility triggered a Complete Response Letter in September 2025, with the US FDA identifying no other approvability issues at the time. Scholar Rock subsequently resubmitted the BLA in March 2026 after adding the alternate fill-finish site. The company said commercial supply from that facility is available if apitegromab is approved.

The manufacturing issue has had a greater impact in Europe. Scholar Rock withdrew its Marketing Authorisation Application through a Committee for Medicinal Products for Human Use written procedure completed August 20 and plans to resubmit using the alternate facility. The EMA had been awaiting resolution of the Catalent Indiana inspection status before issuing an opinion.

Scholar Rock is also preparing to expand the regulatory program into Japan. The Pharmaceuticals and Medical Devices Agency agreed that no additional Japanese clinical studies will be required for a Japanese New Drug Application, allowing the company to target a submission by the end of 2026.

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Apitegromab is a fully human monoclonal antibody designed to inhibit myostatin activation and improve muscle function. Scholar Rock is developing the drug as an add-on to SMN-directed SMA therapies, including nusinersen, risdiplam, and onasemnogene abeparvovec.

The BLA is supported by the Phase III SAPPHIRE trial, in which apitegromab produced a statistically significant 1.8-point placebo-adjusted improvement on the Hammersmith Functional Motor Scale Expanded at 52 weeks in patients aged 2–12 receiving existing SMN-targeted therapy.

Apitegromab could become the first muscle-targeted therapy approved for SMA. Roche has discontinued development of emugrobart (GYM329), another anti-myostatin antibody previously in clinical development for the disease, while Biogen is advancing next-generation SMN-directed approaches including salanersen.


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