Scholar Rock Sets September 30 FDA Decision Date for Apitegromab

Scholar Rock faces a regulatory milestone for its drug candidate, apitegromab, as the FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. The company is moving forward with the muscle-targeted therapy for spinal muscular atrophy (SMA) after addressing manufacturing compliance issues that delayed the review.

"The FDA made clear that the only approvability issue was the state of compliance at the Catalent Indiana fill-finish facility, which is owned by Novo Nordisk," said David Hallal, CEO of Scholar Rock, in a statement at the Cantor Global Healthcare Conference.

Resolving Manufacturing Compliance

To address the regulatory bottleneck, Scholar Rock removed the Catalent Indiana fill-finish site from its Biologics License Application (BLA). The company transitioned to an alternate commercial manufacturing facility, which became operational in the first half of 2026. This shift satisfied FDA requirements regarding the integrity of the production chain.

Global Regulatory Strategy

Scholar Rock is adjusting its international regulatory footprint. The company withdrew its European Marketing Authorization Application in August 2026, choosing to pause those efforts until the FDA reaches a final decision. The firm expects to file for approval with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) by the end of 2026.

Financial Position and Capital Allocation

Scholar Rock holds approximately $500 million in cash. Management reports that this liquidity supports concurrent operations across regulatory filings, commercial launch preparations, and pipeline development.

Understanding the PDUFA Process

The PDUFA date is the deadline for the FDA to complete its review of a drug application. When a company encounters a complete response letter or a manufacturing-related delay, such as the one Scholar Rock faced with the Catalent site, the agency requires a resubmission addressing specific deficiencies. By switching manufacturing partners, Scholar Rock intends to provide the FDA with a verified supply chain, a strategy used by biotech firms to avoid the re-inspection timelines associated with third-party facilities.