Scholar Rock’s ISEMBYLD Secures FDA Approval as First-of-Its-Kind Muscle Therapy for SMA

Scholar Rock (SRRK) announced that the U.S. Food and Drug Administration (FDA) has approved ISEMBYLD (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children aged two and older, provided they are currently undergoing a survival motor neuron 2 (SMN2)-targeted therapy.

Spinal muscular atrophy is a rare, severe neuromuscular disorder marked by irreversible motor neuron loss and progressive muscle wasting. This condition causes continuous motor function decline throughout a patient’s life, profoundly impacting independence for both adults and children.

ISEMBYLD stands as the first and only muscle-targeted therapy demonstrated to enhance motor function in SMA patients already receiving SMN2-targeted treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE clinical trial, patients administered ISEMBYLD achieved robust, clinically significant improvements in motor function after one year of treatment, whereas those relying solely on SMN2-targeted therapy experienced further motor function deterioration.

SRRK closed Friday’s regular trading session at $55.41, down $0.26 or 0.47%. However, in after-hours trading, the stock surged $6.56, or 11.84%.

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