Issuance of Priority Review Voucher; Rare Pediatric Disease Product
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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 ELEVIDYS (delandistrogene moxeparvovec-rokl), manufactured by Sarepta Therapeutics, Inc., meets the criteria for a priority review voucher.
Full Text
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<title>Federal Register, Volume 88 Issue 135 (Monday, July 17, 2023)</title>
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[Federal Register Volume 88, Number 135 (Monday, July 17, 2023)]
[Notices]
[Pages 45430-45431]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[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 <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
ELEVIDYS, 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: 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
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