[Federal Register Volume 89, Number 76 (Thursday, April 18, 2024)]
[Notices]
[Page 27767]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-08276]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-N-1636]


Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product; LENMELDY (Atidarsagene 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 LENMELDY (atidarsagene autotemcel), approved on March 18, 2024, 
manufactured by Orchard Therapeutics (Europe) Ltd., meets the criteria 
for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 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 LENMELDY (atidarsagene autotemcel), 
manufactured by Orchard Therapeutics (Europe) Ltd., meets the criteria 
for a priority review voucher.
    LENMELDY (atidarsagene autotemcel) is indicated for treatment of 
children with pre-symptomatic late infantile, pre-symptomatic early 
juvenile, or early symptomatic early juvenile metachromatic 
leukodystrophy.
    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 
LENMELDY (atidarsagene 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.

    Dated: April 15, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-08276 Filed 4-17-24; 8:45 am]
BILLING CODE 4164-01-P