Issuance of Priority Review Voucher; Rare Pediatric Disease Product; LENMELDY (Atidarsagene Autotemcel)
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Issuing agencies
Abstract
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.
Full Text
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<title>Federal Register, Volume 89 Issue 76 (Thursday, April 18, 2024)</title>
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[Federal Register Volume 89, Number 76 (Thursday, April 18, 2024)]
[Notices]
[Page 27767]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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 <a href="https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs">https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs</a>. 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 <a href="https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products">https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products</a>.
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
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