Scholar Rock Books First ISEMBYLD Sales After FDA Approval

The Tip Desk · November 12, 2026 · 3 min read

The neuromuscular biopharma company recorded $0.6 million of U.S. product revenue in the third quarter and agreed to sell a priority review voucher for $205 million.

Scholar Rock Holding (SRRK), a global biopharmaceutical company focused on rare neuromuscular diseases, recorded its first product revenue after the U.S. Food and Drug Administration approved ISEMBYLD (apitegromab-mstn) on September 11, 2026.

The company booked $0.6 million of net product revenue from U.S. sales of ISEMBYLD in the three months ended September 30, 2026, compared with no product revenue a year earlier. ISEMBYLD is the first and only muscle-targeted therapy for spinal muscular atrophy and is indicated for adults and children two years of age and older who are receiving an SMN2-targeted treatment. Vials were labeled, packaged, and shipped to the specialty network within a week of approval, and the company said initial patients were infused in October 2026.

Net loss widened to $107.2 million, or $0.81 a share, from a $102.2 million, or $0.90 a share, net loss in the year-ago quarter. Research and development expense was $54.1 million, including $6.4 million of stock-based compensation, compared with $50.5 million a year earlier. Selling, general and administrative expense was $52.5 million, including $11.5 million of stock-based compensation, versus $53.1 million a year earlier. Cost of revenue was $8 thousand; before FDA approval, product costs had been expensed as research and development.

Chairman and Chief Executive Officer David L. Hallal said the quarter was marked by the FDA approval and U.S. launch of ISEMBYLD and that early demand across SMA treatment centers, physicians, and patients reinforced unmet need in the community. A J-code application was submitted to CMS in September for an ISEMBYLD-specific J-code on April 1, 2027. The company said it plans to expand access to ISEMBYLD globally, in up to 50 countries, subject to regulatory approvals.

Cash, cash equivalents, and marketable securities were $413.6 million as of September 30, 2026, down from $492 million as of June 30, 2026. In November, after quarter-end, Scholar Rock entered a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher, awarded following the September 2026 FDA approval, for $205 million. Cash is to be received upon closing, subject to customary conditions including the Hart-Scott-Rodino waiting period.

In Europe, the company resubmitted the apitegromab Marketing Authorisation Application for SMA in October 2026 after EMA guidance; the submission was validated and the company expects a Committee for Medicinal Products for Human Use opinion in the first half of 2027. The resubmitted MAA uses the alternate fill-finish facility that supported the U.S. approval, in place of the Catalent Indiana site. In Japan, the apitegromab New Drug Application was submitted and accepted under priority review, with the PMDA typically targeting about a nine-month cycle for priority-review applications; the product also received an Orphan Drug Designation.

Clinical work continued on subcutaneous apitegromab, Phase 2 OPAL in infants and toddlers under two, and Phase 2 FORGE in facioscapulohumeral muscular dystrophy, which is expected to enroll about 60 patients. In September 2026 the FDA granted Fast Track and Orphan Drug designations to apitegromab for FSHD. A Phase 1 study of SRK-439 in healthy volunteers is ongoing; the company expects topline data by year-end 2026 and plans to share the results in early 2027.