Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KRESLADI (marnetegragene autotemcel)
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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 KRESLADI (marnetegragene autotemcel), approved March 26, 2026, manufactured by Rocket Pharmaceuticals, Inc., meets the criteria for a priority review voucher.
Full Text
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<title>Federal Register, Volume 91 Issue 63 (Thursday, April 2, 2026)</title>
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[Federal Register Volume 91, Number 63 (Thursday, April 2, 2026)]
[Notices]
[Page 16714]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-06379]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2026-N-3241]
Issuance of Priority Review Voucher; Rare Pediatric Disease
Product; KRESLADI (marnetegragene autotemcel)
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 KRESLADI (marnetegragene autotemcel), approved March 26, 2026,
manufactured by Rocket Pharmaceuticals, 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#fe97909a8b8d8a8c87d09c9791929199979d8dbe989a9fd096968dd0999188"><span class="__cf_email__" data-cfemail="127b7c76676166606b3c707b7d7e7d757b7161527476733c7a7a613c757d64">[email 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 KRESLADI (marnetegragene autotemcel),
manufactured by Rocket Pharmaceuticals, Inc., meets the criteria for a
priority review voucher. KRESLADI (marnetegragene autotemcel) is
indicated for the treatment of pediatric patients withsevere leukocyte
adhesion deficiency-I (LAD-I) due to biallelic variants inITGB2without
an available human leukocyte antigen (HLA)-matched sibling donor for
allogeneic hematopoietic stem cell transplant.
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
KRESLADI (marnetegragene 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. 2026-06379 Filed 4-1-26; 8:45 am]
BILLING CODE 4164-01-P
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