FDA Approves ISEMBYLD™ (apitegromab-mstn) for Treatment of SMA

Scholar Rock announced that ISEMBYLD™ (apitegromab-mstn) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children 2 years of age and older who are currently receiving an SMN2-targeted treatment. ISEMBYLD showed a clinically meaningful improvement in motor function in the Phase 3 SAPPHIRE clinical trial when added to an SMN2-targeted treatment.

ISEMBYLD is the first and only muscle-targeted treatment approved for SMA.

For more information on the approval, please see the press release from Scholar Rock.