Scholar Rock's Apitegromab Secures FDA Fast Track in FSHD and Commences Phase 2 FORGE Trial Amid Pending SMA Review
Scholar Rock has executed a major expansion of its anti-myostatin pipeline while progressing toward its critical FDA regulatory decision in spinal muscular atrophy (SMA). On September 2, 2026, the company announced that the U.S. Food and Drug Administration (FDA) has granted both Fast Track and Orphan Drug designations to its investigational monoclonal antibody, apitegromab, for the treatment of people living with Facioscapulohumeral Muscular Dystrophy (FSHD). Concurrently, participant dosing has officially commenced in the Phase 2 FORGE clinical trial evaluating the drug in adults with FSHD.
This clinical launch and regulatory fast-tracking significantly diversify Scholar Rock's pipeline, which is anchored by its pending SMA Biologics License Application (BLA) under review for a September 30, 2026 PDUFA action date.
Expanding into FSHD: The FORGE Trial Design
Facioscapulohumeral muscular dystrophy (FSHD) is a rare, severe, and progressive genetic muscle-weakening disease that primarily affects the face, shoulders, and upper arms, often leading to profound physical disability. There are currently no FDA-approved disease-modifying treatments for FSHD.
Apitegromab is a highly selective fully human monoclonal antibody designed to inhibit myostatin activation by binding to its precursor (pro- and latent) forms in skeletal muscle, thereby promoting muscle growth and strength. The FSHD program is supported by translational preclinical data in the gold-standard FLExDUX4 mouse model, where a murine version of apitegromab demonstrated significant increases in muscle mass, strength, and endurance.
The newly initiated FORGE trial (NCT07435129) is a Phase 2 randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of apitegromab as a monotherapy in adults with genetically confirmed FSHD.
Pending SMA Regulatory Milestone
Apitegromab's primary near-term catalyst remains its BLA for the treatment of spinal muscular atrophy (SMA), which has a target PDUFA date of September 30, 2026.
As part of its regulatory strategy to de-risk the FDA's review, Scholar Rock previously amended its BLA to remove the troubled Catalent Indiana fill-finish facility, relying instead on its second, fully operational fill-finish CDMO. This proactive operational pivot was accepted by the FDA and did not trigger any extension of the PDUFA timeline, keeping the company on track for a potential commercial launch in late 2026.
"Scholar Rock today announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for the treatment of people living with FSHD. In addition, the Company announced that participant dosing is underway in the Phase 2 FORGE clinical trial evaluating apitegromab... in people living with FSHD." — Scholar Rock Press Release (September 2, 2026)
"Apitegromab therefore holds important potential to impact this disease... participant dosing is underway in the Phase 2 FORGE clinical trial evaluating apitegromab, an investigational fully human monoclonal antibody designed to inhibit myostatin activation, in people living with FSHD." — Akshay Vaishnaw, M.D., Ph.D., President of R&D at Scholar Rock