FDA Grants Fast Track and Orphan Drug Status to Apitegromab for FSHD as Phase 2 Dosing Begins
SRRK has more than doubled off its 52-week low of $27.07.
Summary
Scholar Rock announced FDA Fast Track and Orphan Drug designations for apitegromab in facioscapulohumeral muscular dystrophy (FSHD), and dosing has begun in the Phase 2 FORGE trial. FSHD has no approved therapies, so these designations validate the program's potential and could expedite development. The FORGE trial will enroll about 60 patients and read out at Week 52, with a key secondary endpoint at Week 24. This expands apitegromab beyond SMA, where a PDUFA decision is due September 30, 2026. The news adds a second late-stage opportunity for the company's myostatin platform.
At the time of this announcement, SRRK was trading at $55.98 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $6.8B. The 52-week trading range was $27.07 to $61.76. This news item was assessed with positive market sentiment and an importance score of 7 out of 10. Source: BusinessWire.