SCHOLAR ROCK HOLDING CORP · Data as of 2026-09-08
2026
Positive data from the successful Phase 3 SAPPHIRE study evaluating apitegromab in children and adults with SMA were reported in October 2024, and regulatory approvals are anticipated in the U.S. and Europe in 2026.Source filing
late 2026
A Phase 1 study evaluating SRK-439 in healthy volunteers is progressing well with topline data anticipated in late 2026.Source filing
Sep 30
Apitegromab Biologics License Application (BLA) on track for potential FDA approval by September 30, 2026 Prescription Drug User Fee Act (PDUFA) date.Source filing