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, ...
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.