On September 11, 2026, the FDA approved Scholar Rock’s ISEMBYLD (apitegromab-mstn) for spinal muscular atrophy (SMA).
The drug is approved for adults and children ages 2 and older who are already receiving an SMN2-targeted SMA treatment.
ISEMBYLD is designed to target muscle loss directly, complementing existing therapies that target motor neurons. It is described as the first muscle-targeted treatment for SMA.
Approval was supported by late-stage data showing a statistically significant improvement in motor function:
patients receiving the recommended dose improved by 2.2 points on the HFMSE scale versus placebo.
The company said U.S. commercial launch preparations were underway, with product shipments expected within days. The approval marks Scholar Rock’s first regulatory approval.
Scholar Rock’s shares reportedly rose in after-hours trading following the decision.
Sources:
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