FDA approves Scholar Rock's ISEMBYLD (apitegromab) for SMA in adults and children ≥2 years

Scholar Rock Holding Corp

• FDA approved ISEMBYLD (apitegromab-mstn) for SMA in adults/children ≥2 yrs on SMN2-targeted therapy.
• Phase 3 SAPPHIRE: 2.2-point HFMSE improvement vs placebo (nominal p=0.0121); 34.2% ≥3-point gain vs 13.5% (OR 3.8).
• Safety: fractures 9% ISEMBYLD vs 2% placebo; database >500, some treated >7 years.
• U.S. launch underway, shipping in coming days; awarded Rare Pediatric Disease Priority Review Voucher.