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

Issuing agencies

Abstract

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. 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.

Full Text

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<title>Federal Register, Volume 91 Issue 192 (Tuesday, October 6, 2026)</title>
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[Federal Register Volume 91, Number 192 (Tuesday, October 6, 2026)]
[Notices]
[Page 63569]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-20434]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2026-N-11259]


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

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
issuance of a priority review voucher to the sponsor of a rare 
pediatric disease product application. 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, 
<a href="/cdn-cgi/l/email-protection#422b2c26373136303b6c202b2d2e2d252b2131022426236c2a2a316c252d34"><span class="__cf_email__" data-cfemail="95fcfbf1e0e6e1e7ecbbf7fcfaf9faf2fcf6e6d5f3f1f4bbfdfde6bbf2fae3">[email&#160;protected]</span></a>, 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 
with preserved neurodevelopmental function.
    For further information about the Rare Pediatric Disease Priority 
Review Voucher Program and for a link to the full text of section 529 
of the FD&C Act, go to <a href="https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs">https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs</a>. For further information about FAYUVI 
(rebisufligene etisparvovec-hopf), go to the Center for Biologics 
Evaluation and Research's Approved Cellular and Gene Therapy Products 
website at <a href="https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products">https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products</a>.

Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-20434 Filed 10-5-26; 8:45 am]
BILLING CODE 4164-01-P


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