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On September 11, 2026, the U.S. Food and Drug Administration approved ISEMBYLD (apitegromab-mstn) for the treatment of…
SRRKScholar Rock Holding Corp
On September 11, 2026, the U.S. Food and Drug Administration approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy in adults and children two years of age and older currently receiving SMN2-targeted treatment. The approval was based on Phase 3 SAPPHIRE study results showing a 2.2-point improvement in Hammersmith Functional Motor Scale-Expanded scores compared to placebo. Scholar Rock is launching ISEMBYLD commercially in the U.S., with product available for shipment immediately, and received a Rare Pediatric Disease Priority Review Voucher. The company reported $492 million in cash, cash equivalents, and marketable securities as of June 30, 2026, and intends to monetize the voucher. Scholar Rock Holding Corporation is a global biopharmaceutical company focused on developing treatments for rare neuromuscular diseases.