Issuance of Priority Review Voucher; Rare Pediatric Disease Product; FAYUVI (rebisufligene etisparvovec-hopf)

The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that FAYUVI (rebisufligene etisparvovec-hopf), approved on September 17, 2026, manufactured by Ultragenyx Pharmaceutical Inc., meets the criteria for a priority review voucher. FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics Evaluation and Research, Food and Drug Administration, [email protected] , 240-402-7911. SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority review voucher to the sponsor of an approved rare pediatric disease product application. Under section 529 of the FD&C Act (21 U.S.C. 360ff), FDA will award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined that FAYUVI (rebisufligene etisparvovec-hopf), manufactured by Ultragenyx Pharmaceutical Inc., meets the criteria for a priority review voucher. FAYUVI (rebisufligene etisparvovec-hopf) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients wit