Issuance of Priority Review Voucher; Rare Pediatric Disease Product; WASKYRA (etuvetidigene autotemcel)
Primary source
Metadata and text below are from the Federal Register, a public-domain U.S. government work. Always verify the official published version before relying on it for any legal matter.
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 WASKYRA (etuvetidigene autotemcel), approved on December 9, 2025, manufactured by Fondazione Telethon ETS, meets the criteria for a priority review voucher.
Full Text
<html>
<head>
<title>Federal Register, Volume 91 Issue 35 (Monday, February 23, 2026)</title>
</head>
<body><pre>
[Federal Register Volume 91, Number 35 (Monday, February 23, 2026)]
[Notices]
[Page 8494]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-03493]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2026-N-0672]
Issuance of Priority Review Voucher; Rare Pediatric Disease
Product; WASKYRA (etuvetidigene autotemcel)
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
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 WASKYRA (etuvetidigene autotemcel), approved on December 9, 2025,
manufactured by Fondazione Telethon ETS, meets the criteria for a
priority review voucher.
FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 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 WASKYRA (etuvetidigene autotemcel),
manufactured by Fondazione Telethon ETS, meets the criteria for a
priority review voucher. WASKYRA (etuvetidigene autotemcel) is
indicated for the treatment of pediatric patients aged 6 months and
older and adults with Wiskott-Aldrich Syndrome (WAS) who have a
mutation in the WAS gene and for whom hematopoietic stem cell
transplantation (HSCT) is appropriate and no suitable human leukocyte
antigen (HLA)-matched related stem cell donor is available.
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 WASKYRA
(etuvetidigene autotemcel), 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. 2026-03493 Filed 2-20-26; 8:45 am]
BILLING CODE 4164-01-P
</pre></body>
</html>This is legal information, not legal advice. Laws vary by jurisdiction and change frequently. Always verify current law with official sources and consult a licensed attorney in your jurisdiction for advice on your specific situation.