$SRRK (SCHOLAR ROCK HOLDING) — 8-K: Scholar Rock wins FDA approval for ISEMBYLD, first muscle-targeted SMA treatment

The FDA approved ISEMBYLD (apitegromab-mstn) for SMA patients aged 2 and older already on an SMN2-targeted treatment. In the Phase 3 SAPPHIRE study, the 10 mg/kg dose produced a 2.2-point HFMSE motor function improvement versus placebo (p = 0.0121), and 34.2% of treated patients gained 3 points versus 13.5% on placebo (odds ratio 3.8; p = 0.0125). U.S. launch is underway with product shipping in coming days.

The FDA approved ISEMBYLD (apitegromab-mstn) for SMA patients aged 2 and older already on an SMN2-targeted treatment. In the Phase 3 SAPPHIRE study, the 10 mg/kg dose produced a 2.2-point HFMSE motor