Apitegromab - (SAPPHIRE)

August 21, 2026: Scholar Rock (NASDAQ: SRRK) said the FDA-guided removal of Catalent Indiana as the commercial fill-finish site from the apitegromab BLA for spinal muscular atrophy is complete, and FDA review is continuing with an alternate U.S. fill-finish facility. The company said it remains prepared for a U.S. launch upon potential approval, with a September 30, 2026 PDUFA date and commercial vial supply available from the alternate site.

In Europe, after FDA classified Catalent Indiana’s April 2026 inspection as Official Action Indicated and notified customers on August 7, 2026, Scholar Rock withdrew the apitegromab MAA through a CHMP written procedure that concluded August 20, 2026. It plans to resubmit the MAA with the alternate fill-finish facility to speed the path to CHMP opinion.

In Japan, Scholar Rock said PMDA agreed no additional Japanese clinical studies are required for filing, based on guidelines allowing certain orphan/rare disease applications without Japanese patient data. The company is now targeting submission of a Japanese NDA for apitegromab in children and adults with SMA by year-end 2026.