FDA approves ISEMBYLD for spinal muscular atrophy treatment

spinal muscular atrophy SMA ISEMBYLD muscle-targeted therapy FDA approval

The FDA has approved ISEMBYLD (apitegromab-mstn) for treating spinal muscular atrophy (SMA) in adults and children aged two years and older who are already receiving survival motor neuron 2 (SMN2)-targeted treatment.

Developed by Scholar Rock, ISEMBYLD is the first muscle-targeted therapy for SMA, a neuromuscular disease characterized by progressive muscle wasting and loss of motor neurons. In clinical trials, a 10 mg/kg dose demonstrated a 2.2-point improvement in motor function on the Hammersmith Functional Motor Scale-Expanded compared to a placebo.

Scholar Rock has initiated the U.S. commercial launch, with the product expected to ship in the coming days.

Entities

FDA · ISEMBYLD · Scholar Rock

Sources

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