[Federal Register Volume 88, Number 135 (Monday, July 17, 2023)]
[Notices]
[Pages 45430-45431]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-15079]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2023-N-2607]


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 (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 ELEVIDYS (delandistrogene moxeparvovec-rokl), manufactured by 
Sarepta Therapeutics, Inc., 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.

[[Page 45431]]


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 ELEVIDYS, manufactured by Sarepta Therapeutics, 
Inc., meets the criteria for a priority review voucher.
    ELEVIDYS is indicated for treatment of ambulatory pediatric 
patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) 
with confirmed mutation in the DMD gene.
    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 
ELEVIDYS, 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: July 12, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-15079 Filed 7-14-23; 8:45 am]
BILLING CODE 4164-01-P