Scholar Rock's ISEMBYLD Wins FDA Approval As First Muscle-Targeted Therapy For SMA

(RTTNews) - Scholar Rock (SRRK) announced that the U.S. Food and Drug Administration approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted therapy.

Spinal muscular atrophy or SMA is a rare, severe neuromuscular disease characterized by irreversible loss of motor neurons and progressive muscle wasting. The condition leads to continuous motor function decline throughout life, severely impacting independence for both adults and children.

ISEMBYLD is the first and only muscle-targeted therapy shown to improve motor function in individuals with SMA who are already on SMN2-targeted treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE study, patients receiving ISEMBYLD demonstrated robust, clinically meaningful motor function improvement after one year of treatment, while those on SMN2-targeted therapy alone experienced further motor function loss.

SRRK closed Friday's regular trading at $55.41 down $0.26 or 0.47%. But in the after-hours trading the stock gained $6.56 or 11.84%.

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