Scholar Rock reports first ISEMBYLD sales, agrees to sell $205 million PRV

Scholar Rock said ISEMBYLD (apitegromab-mstn) was approved by the FDA on September 11, 2026, as the first and only muscle-targeted therapy for SMA, indicated for adults and children two years of age and older who are currently receiving an SMN2-targeted treatment.¹ The company reported ISEMBYLD vials were labeled, packaged, and shipped to the specialty network within one week of the FDA approval¹, and a J-code application was submitted to the Centers for Medicare & Medicaid Services in September, seeking an ISEMBYLD-specific J-code effective April 1, 2027.¹ The drug generated net product revenue of $0.6 million from U.S. sales in the quarter ended September 30, 2026.¹

On the regulatory front, Scholar Rock resubmitted the apitegromab Marketing Authorisation Application to the European Medicines Agency in October 2026, and following validation the company expects a CHMP opinion in the first half of 2027.¹ In Japan, the company submitted the apitegromab Japanese New Drug Application, which was accepted under priority review, a process PMDA has typically targeted for completion in about nine months.¹

In its pipeline, the Phase 2 OPAL trial has advanced enrollment and ongoing dosing (NCT07047144), studying apitegromab in infants and toddlers under two years old who received an SMN1-targeted gene therapy or are on an SMN2-targeted therapy.¹ The Phase 2 FORGE trial in facioscapulohumeral muscular dystrophy continues enrollment, with the randomized, placebo-controlled study expected to enroll about 60 patients, measuring mean lean muscle volume change from baseline at 12 months as its primary endpoint.¹ Separately, a Phase 1 study of SRK-439 in healthy volunteers is progressing, with topline data expected by year-end 2026.¹

On financing, Scholar Rock entered into an asset purchase agreement on November 9, 2026, with BioNTech US Inc. to sell its Rare Pediatric Disease Priority Review Voucher for $205 million in cash upon closing.¹ The company reported cash, cash equivalents, and marketable securities of $413.6 million as of September 30, 2026.¹