Apitegromab - (SAPPHIRE)

August 7, 2026: Scholar Rock (NASDAQ: SRRK) said the FDA review of its apitegromab BLA for children and adults with spinal muscular atrophy remains on track for the September 30, 2026 PDUFA date, but the company will remove Catalent Indiana from the filing after being notified that FDA classified the April 2026 inspection of that fill-finish site as Official Action Indicated (OAI).

The BLA, submitted in March 2026, originally included two fill-finish facilities. FDA review will now proceed solely with a second U.S.-based fill-finish facility. Scholar Rock said a data package for FDA review of the second facility, agreed at a March 2026 Type C meeting, was submitted ahead of schedule and review is progressing. The company also said it has commercial vial supply from the second facility available for packaging and labeling and is prepared for a U.S. launch immediately upon approval.

For Europe, Scholar Rock said it is working with EMA on next steps to add the second fill-finish facility to the apitegromab MAA and will provide updated timing for a CHMP opinion once aligned with the agency.