FDA Approves ISEMBYLD for Spinal Muscular Atrophy, First Muscle-Targeted Treatment

Scholar Rock announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children aged two years and older who are currently receiving an SMN2-targeted treatment. This is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA already on SMN2-targeted therapy.