FDA Approves ISEMBYLD for Spinal Muscular Atrophy (SMA)

Scholar Rock announced that the FDA has 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 treatment. This represents the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA already on an SMN2-targeted therapy.