Document ID: FDA-2020-N-0026-0022
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2023-04-24T04:00Z

[Federal Register Volume 88, Number 78 (Monday, April 24, 2023)]
[Notices]
[Pages 24806-24807]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-08518]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-N-0026]

Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product

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 (the FD&C Act), as amended by the Food and Drug

[[Page 24807]]

Administration Safety and Innovation Act (FDASIA), 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 JOENJA (leniolisib), approved March 24, 2023, 
and manufactured by Pharming Technologies B.V., 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, email: 
[email protected].

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), which was added by FDASIA, FDA will award priority review 
vouchers to sponsors of approved rare pediatric disease product 
applications that meet certain criteria. FDA has determined that JOENJA 
(leniolisib), manufactured by Pharming Technologies B.V., meets the 
criteria for a priority review voucher.
    JOENJA (leniolisib) is a kinase inhibitor indicated for the 
treatment of activated phosphoinositide 3-kinase delta (PI3K[delta]) 
syndrome (APDS) in adult and pediatric patients 12 years of age or 
older.
    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 JOENJA (leniolisib), go to the ``[email protected]'' website 
at https://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: April 18, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-08518 Filed 4-21-23; 8:45 am]
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