Priority Review Voucher Issuance; KRESLADI Rare Pediatric Disease Product
Summary
FDA announced the issuance of a priority review voucher to Rocket Pharmaceuticals, Inc. for KRESLADI (marnetegragene autotemcel), approved March 26, 2026, for treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I). The voucher was awarded under section 529 of the FD&C Act's Rare Pediatric Disease Priority Review Voucher Program. This notice fulfills FDA's statutory requirement to publish voucher awards.
What changed
FDA issued a priority review voucher to Rocket Pharmaceuticals for KRESLADI (marnetegragene autotemcel), a gene therapy for severe leukocyte adhesion deficiency-I (LAD-I) in pediatric patients. The product was approved on March 26, 2026, and FDA has determined it meets the criteria for a priority review voucher under section 529 of the FD&C Act (21 U.S.C. 360ff). The voucher allows the sponsor to receive priority review for a future drug application.
This notice is informational and creates no new obligations for the pharmaceutical industry. Other sponsors developing rare pediatric disease products should be aware of the program requirements for earning priority review vouchers. No immediate action is required by regulated entities, and no compliance deadlines apply to this notice.
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Apr 2, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Notice
Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KRESLADI (marnetegragene autotemcel)
A Notice by the Food and Drug Administration on 04/02/2026
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- Public Inspection Published Document: 2026-06379 (91 FR 16714) Document Headings ###### Department of Health and Human Services
Food and Drug Administration
- [Docket No. FDA-2026-N-3241]
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 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, industry.biologics@fda.hhs.gov, 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 in ITGB2 without 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 https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs. 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 https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.
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
Published Document: 2026-06379 (91 FR 16714)
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