FDA Awards Priority Review Voucher for SMA Treatment ISEMBYLD

The U.S. Food and Drug Administration has granted a priority review voucher to Scholar Rock, Inc. for ISEMBYLD (apitegromab-mstn), a treatment for spinal muscular atrophy (SMA). This voucher, issued under the Rare Pediatric Disease program, follows the drug's approval on September 11, 2026, for patients aged two and older already receiving SMN2-targeted therapy. The program aims to incentivize the development of therapies for rare pediatric conditions, highlighting the importance of new treatments for SMA patients.