2024-08276. Issuance of Priority Review Voucher; Rare Pediatric Disease Product; LENMELDY (Atidarsagene Autotemcel)

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

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.