FDA Issues Priority Review Voucher for KRESLADI (marnetegragene autotemcel) – Rare Pediatric Disease Product
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The U.S. Food and Drug Administration (FDA) has formally announced the issuance of a Priority Review Voucher (PRV) to Rocket Pharmaceuticals, Inc., the sponsor of KRESLADI (marnetegragene autotemcel), a gene therapy approved on March 26, 2026 for a rare pediatric disease. The PRV program, established under the Federal Food, Drug, and Cosmetic Act (FD&C Act), incentivizes pharmaceutical companies to develop treatments for rare pediatric conditions by awarding a transferable voucher that can be used to obtain an expedited FDA review for a future drug application. KRESLADI is a gene therapy product and represents a significant milestone in rare disease treatment. Rocket Pharmaceuticals earned this voucher by successfully obtaining FDA approval for a qualifying rare pediatric disease product, fulfilling all statutory criteria under the PRV program. FDA is legally required to publish public notice upon awarding such vouchers. Priority Review Vouchers are highly valuable in the pharmaceutical industry as they can be sold or transferred to other companies. When redeemed, a PRV reduces the FDA's standard review timeline from 10 months to 6 months for the designated application, offering a significant competitive and commercial advantage to the holder.
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Key Changes
- FDA approved KRESLADI (marnetegragene autotemcel) on March 26, 2026 for a rare pediatric disease indication
- Rocket Pharmaceuticals, Inc. is awarded a transferable Priority Review Voucher (PRV) under the FD&C Act
- The PRV entitles the holder to request expedited FDA review (6 months instead of standard 10 months) for any future drug application
+ 2 more changes with Pro
Obligations
What this law requires
FDA must publish public notice upon awarding a Priority Review Voucher for rare pediatric disease products
Sponsor must obtain FDA approval for a rare pediatric disease product to qualify for a Priority Review Voucher
FDA must verify that approved rare pediatric disease products meet statutory criteria under the PRV program before awarding vouchers
PRV holder may transfer or sell the Priority Review Voucher to another entity
PRV holder must designate a single future drug application to receive expedited 6-month FDA review when redeeming the voucher