Scholar Rock Holding Corporation announced that the U.S. Food and Drug Administration (FDA) has approved ISEMBYLD™ (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA). It is the first muscle-targeted therapy approved for adults and children aged 2 and older who are currently receiving an SMN2-targeted treatment. The company is proceeding with an immediate U.S. commercial launch.
Key Details
- Indication: Approved as a treatment for spinal muscular atrophy (SMA) in adults and children (2 years and older) who are also on an existing SMN2-targeted therapy.
- Clinical Efficacy: The approval is based on the Phase 3 SAPPHIRE study, which demonstrated a statistically significant 2.2-point improvement in the HFMSE motor function scale compared to placebo (p=0.0121).
- Commercialization: The U.S. commercial launch is underway, with the product expected to be available for shipment within days. The company also received a Rare Pediatric Disease Priority Review Voucher, which can be used for a future application or monetized.