FDA Hands Drugmaker a Priority Voucher for Rare-Disease Treatment
Published Date: 10/26/2026
Notice
Summary
The FDA just gave Scholar Rock, Inc. a special priority review voucher for their new rare pediatric disease drug, ISEMBYLD, approved on September 11, 2026. This drug helps treat spinal muscular atrophy in kids and adults already on certain treatments. The voucher speeds up future FDA reviews, saving time and potentially money for the company and encouraging more rare disease drug development.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
ISEMBYLD Approved for SMA Patients 2+
ISEMBYLD (apitegromab-mstn) was approved by the FDA on September 11, 2026 and is indicated to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. The approval and indication are explicitly stated in the FDA notice.
Priority Review Voucher Issued to Sponsor
The FDA issued a priority review voucher to the sponsor of ISEMBYLD (apitegromab-mstn), manufactured by Scholar Rock, Inc. The notice says ISEMBYLD was approved on September 11, 2026 and that the voucher was awarded under section 529 (21 U.S.C. 360ff) of the Federal Food, Drug, and Cosmetic Act.
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