FDA Approves Isembyld, First Muscle-Targeted Therapy for Spinal Muscular Atrophy

The U.S. Food and Drug Administration (FDA) has approved Isembyld (apitegromab-mstn), a monthly infusion treatment for individuals with spinal muscular atrophy (SMA) who are already receiving SMN2-targeted therapies. This approval represents the first and only muscle-targeted therapy for SMA, offering new hope for children and adults living with the neuromuscular disease.