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Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)

SRRK
  • Scholar Rock resubmitted the Biologics License Application (BLA) for apitegromab to the FDA on March 31, 2026, following a March 3, 2026 Type C meeting.
  • The resubmission includes Catalent Indiana LLC (part of Novo Nordisk) & a second U.S.-based fill-finish facility to strengthen the supply chain.
  • FDA & Scholar Rock aligned in Q1 2026 to resubmit prior to a reinspection of the Catalent Indiana facility, which had been subject to observations in September 2025 that did not relate specifically to apitegromab.
  • The company anticipates FDA acceptance of the BLA within 30 days of resubmission & a review period of up to six months.
  • A PDUFA action date is expected in late September 2026.
  • Updates to the BLA were limited in scope & primarily composed of a standard safety update.
  • The European Medicines Agency (EMA) review of the Marketing Authorisation Application (MAA) is progressing well, with a decision anticipated in mid-2026.
  • A conference call to discuss the resubmission is scheduled for March 31, 2026, at 8:00 a.m. ET.
  • Apitegromab is the first muscle-targeted therapy to show statistically significant motor function improvements in a Phase 3 trial for spinal muscular atrophy (SMA).
  • The FDA has granted Fast Track, Orphan Drug, Priority Review, & Rare Pediatric Disease designations for apitegromab.
  • The EMA has granted Priority Medicines (PRIME) & Orphan Medicinal Product designations for apitegromab.