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Issuance of Priority Review Voucher; Rare Pediatric Disease Product; AQNEURSA (Levacetylleucine)

89 Fed. Reg. 83023 (Oct. 15, 2024)

Issuance of Priority Review Voucher; Rare Pediatric Disease Product; AQNEURSA (Levacetylleucine)

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Federal RegisterOct 15, 2024
89 Fed. Reg. 83023 (Oct. 15, 2024)
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    Department of Health and Human Services Food and Drug Administration
  • [Docket No. FDA-2020-N-0026]

    • 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 AQNEURSA (levacetylleucine), approved on September 24, 2024, manufactured by IntraBio Inc., meets the criteria for a priority review voucher.

    FOR FURTHER INFORMATION CONTACT:

    Cathryn Lee, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-1394.

    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 AQNEURSA (levacetylleucine), manufactured by IntraBio Inc., meets the criteria for a priority review voucher. AQNEURSA (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg.

    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/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further information about AQNEURSA (levacetylleucine), go to the “Drugs@FDA” website at http://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: October 8, 2024.

    Eric Flamm,

    Acting Associate Commissioner for Policy.

    [FR Doc. 2024-23712 Filed 10-11-24; 8:45 am]

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