FDA Grants Priority Review Voucher for Rare Pediatric Drug
Published Date: 5/11/2026
Notice
Summary
The FDA just gave Denali Therapeutics a special priority review voucher for their rare pediatric disease drug AVLAYAH, approved in March 2026. This voucher speeds up the review of future medicines, helping kids with rare diseases get treatments faster. It’s a big win for patients, the company, and anyone watching rare disease drug development!
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
New FDA Approval for Hunter Syndrome Pediatrics
AVLAYAH (tividenofusp alfa-eknm) was approved March 24, 2026 for the treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II). The approval applies when treatment is started in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment.
Priority Review Voucher Awarded to Sponsor
The FDA issued a priority review voucher to Denali Therapeutics Inc. for AVLAYAH (tividenofusp alfa-eknm); AVLAYAH was approved March 24, 2026. The voucher was awarded under section 529 of the Federal Food, Drug, and Cosmetic Act.
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