FDA Fast-Tracks Kid Gene Therapy with Special Voucher
Published Date: 10/6/2026
Notice
Summary
The FDA just gave a special priority review voucher to Ultragenyx for their new gene therapy, FAYUVI, which treats a rare childhood disease called Sanfilippo syndrome type A. This voucher speeds up future drug reviews, helping get important medicines to kids faster. The approval happened on September 17, 2026, marking a big win for rare disease treatments and the company behind it.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
New Gene Therapy Approved for Sanfilippo
FAYUVI (rebisufligene etisparvovec-hopf) was approved on September 17, 2026 for treatment of the neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. Pediatric patients meeting that indication now have an FDA-approved gene therapy for this condition.
Priority Review Voucher Awarded
The FDA issued a priority review voucher to Ultragenyx Pharmaceutical Inc. for FAYUVI (rebisufligene etisparvovec-hopf) following the product's approval on September 17, 2026. The award was made under section 529 of the Federal Food, Drug, and Cosmetic Act.
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