Scholar Rock receives FDA approval for Isembyld, first muscle-targeted SMA therapy
Scholar Rock announced that its monoclonal antibody apitegromab, branded Isembyld, has been cleared by the FDA for treatment of spinal muscular atrophy (SMA).
The approval follows a previous rejection in September 2025 due to compliance issues at a third‑party manufacturer, but the agency granted clearance weeks ahead of the original target date of Sept. 30.
Isembyld is the first therapy that directly targets muscle loss in SMA patients aged two years and older who are also receiving SMN2‑targeted treatments, complementing existing options such as Biogen’s Spinraza and Novartis’ Zolgensma.
The company said the drug will be available to ship in the coming days.
This writeup was produced by pharmadog from original reporting by BioSpace.
Original headline: “Scholar Rock secures first FDA approval for muscle-targeting rare disease drug”
read at BioSpace ↗
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