Scholar Rock (SRRK) FDA announcement summary

Introduction and purpose

• FDA approved ISEMBYLD (apitegromab-mstn) as the first muscle-targeted treatment for spinal muscular atrophy (SMA) in adults and children aged 2 and older receiving SMN2-targeted therapy.

• SMA is a rare neuromuscular disease causing progressive muscle wasting and loss of motor function.

Details of approval or decision

• Approval covers all adults and children ≥2 years with SMA on SMN2-targeted treatment.

• ISEMBYLD is the first therapy to directly target muscle in SMA, addressing a significant unmet need.

• A Rare Pediatric Disease Priority Review Voucher was awarded with the approval.

Impact on industry and stakeholders

• Marks a major advance for the SMA community, offering a new therapeutic option to improve motor function.

• Commercial launch in the U.S. is underway, with product shipping in the coming days.

• Dedicated support services are available to assist patients and caregivers with access and logistics.