Federal Register #2026-20434

FDA Announces Priority Review Voucher for Ultragenyx’s FAYUVI Gene Therapy

The U.S. Food and Drug Administration announced a rare pediatric disease priority review voucher for FAYUVI, manufactured by Ultragenyx Pharmaceutical Inc. - Government agency: U.S. Department of Health and Human Services, Food and Drug Administration (FDA). - Award: FDA issued a rare pediatric disease priority review voucher to the sponsor of FAYUVI after determining that the product met applicable criteria. - OEM: Ultragenyx Pharmaceutical Inc. manufactures FAYUVI (rebisufligene etisparvovec-hopf). - Product context: FAYUVI is an adeno-associated virus vector-based gene therapy indicated for neurologic manifestations of mucopolysaccharidosis type IIIA in pediatric patients with preserved neurodevelopmental function. - Procurement status: This is an award announcement, not a solicitation; no products or services are requested for purchase.

Description

The Food and Drug Administration (FDA) announces issuance of a priority review voucher to the sponsor of an approved rare pediatric disease product application. FDA determined that FAYUVI, manufactured by Ultragenyx Pharmaceutical Inc. and approved on September 17, 2026, meets the criteria for a voucher. FAYUVI is an adeno-associated virus vector-based gene therapy indicated to treat neurologic manifestations of mucopolysaccharidosis type IIIA in pediatric patients with preserved neurodevelopmental function.

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