Federal Register #2026-06379

FDA Awards Priority Review Voucher for KRESLADI Rare Pediatric Disease Product

This notice announces the award of a priority review voucher by the Food and Drug Administration (FDA), part of the Department of Health and Human Services, for a rare pediatric disease product. - Government Buyer: - Food and Drug Administration (FDA), Department of Health and Human Services - OEM and Vendor: - Rocket Pharmaceuticals, Inc. (manufacturer and recipient) - Product Awarded: - KRESLADI (marnetegragene autotemcel) - Indicated for pediatric patients with severe leukocyte adhesion deficiency-I (LADI) due to biallelic variants in ITGB2 - Specifically for patients without an available HLA-matched sibling donor for allogeneic hematopoietic stem cell transplant - Notable Requirements: - Award made under the Federal Food, Drug, and Cosmetic Act for approved rare pediatric disease product applications meeting specific criteria - Public announcement of the priority review voucher issuance - No procurement of goods or services is solicited; this is an award announcement

Description

The Food and Drug Administration (FDA) has issued a priority review voucher to Rocket Pharmaceuticals, Inc. for KRESLADI (marnetegragene autotemcel), a rare pediatric disease product approved on March 26, 2026. This voucher is awarded under the Federal Food, Drug, and Cosmetic Act to sponsors of approved rare pediatric disease product applications that meet specific criteria. KRESLADI is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LADI) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. The notice serves to publicly announce the award of this priority review voucher.

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