Scholar Rock resubmits drug application to FDA for SMA treatment

Scholar Rock (NASDAQ: SRRK) has resubmitted its Biologics License Application to the FDA for apitegromab, a treatment for children and adults with spinal muscular atrophy.

The resubmission includes Catalent Indiana LLC, part of Novo Nordisk, and a second U.S.-based fill-finish facility. The company made this decision following guidance from a March 3 FDA Type C meeting and interactions with both the FDA and Novo Nordisk during the first quarter of 2026.

Scholar Rock received a Complete Response Letter from the FDA in September 2025 regarding observations identified during a routine inspection of Catalent Indiana. The observations were not specific to apitegromab, and the letter cited no other approvability concerns.

The company expects the FDA to accept the resubmitted application within 30 days and anticipates a review period of up to six months. A PDUFA action date is expected in late September 2026.

The resubmission updates were limited in scope and primarily included a standard safety update. In Europe, the European Medicines Agency review of the Marketing Authorisation Application for apitegromab continues, with a decision anticipated mid-2026.

Apitegromab has received Fast Track, Orphan Drug, Priority Review, and Rare Pediatric Disease designations from the FDA. The European Medicines Agency has granted Priority Medicines and Orphan Medicinal Product designations.

The drug is described as the first muscle-targeted therapy to demonstrate statistically significant improvements in motor function in a pivotal Phase 3 clinical trial in SMA patients receiving treatment with an SMN-targeted therapy.

Scholar Rock will host a conference call today at 8:00 a.m. ET to discuss the resubmission.

You May Also Be Interested In

• Alphabet added to Dow Jones Industrial Average, Verizon dropped
• Cboe launches binary options suite tied to Mini-S&P 500 Index
• 8x8 reports data breach via third-party Salesforce integration