Scholar Rock targets 2026 apitegromab BLA resubmission and launch

Scholar Rock (NASDAQ: SRRK) announced its 2026 strategic priorities, with plans to resubmit its Biologics License Application for apitegromab and launch the spinal muscular atrophy treatment in the U.S. following FDA approval.

The biopharmaceutical company received a Complete Response Letter from the FDA in September 2025 regarding manufacturing issues at the Catalent Indiana facility. The FDA has scheduled a meeting with Catalent Indiana, now part of Novo Nordisk, in early 2026 to discuss remediation progress at the Bloomington, Indiana fill-finish facility.

Scholar Rock has reserved commercial capacity at a second U.S.-based fill-finish facility beginning in the first quarter of 2026 to strengthen supply continuity. The company expects to submit a supplemental BLA with this second site later in 2026.

The European Medicines Agency continues its regulatory review of apitegromab, with a decision expected on the Marketing Authorisation Application in mid-2026.

The company cleared an Investigational New Drug application for apitegromab in facioscapulohumeral muscular dystrophy and plans to initiate a Phase 2 study called FORGE in mid-2026. Patient dosing is underway in the Phase 2 OPAL trial evaluating apitegromab in infants and toddlers under two years of age with SMA.

Scholar Rock completed a Phase 1 study of subcutaneous apitegromab in healthy volunteers, demonstrating favorable bioavailability with a pharmacodynamic profile comparable to intravenous administration. The company is also conducting a Phase 1 trial of SRK-439, a subcutaneous myostatin inhibitor, with topline data expected in the second half of 2026.

As of December 31, 2025, Scholar Rock reported approximately $365 million in cash, cash equivalents, and marketable securities, expected to fund operations into 2027.

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