[Federal Register Volume 85, Number 15 (Thursday, January 23, 2020)]
[Notices]
[Page 3935]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-01059]
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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 (FD&C Act), as amended by the Food and Drug 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 VYONDYS 53 (golodirsen), manufactured by Sarepta Therapeutics,
Inc., meets the criteria for a priority review voucher.
FOR FURTHER INFORMATION CONTACT: Althea Cuff, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301-
796-9856, 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
VYONDYS 53 (golodirsen), manufactured by Sarepta Therapeutics, Inc.,
meets the criteria for a priority review voucher. VYONDYS 53
(golodirsen) is indicated for the treatment of Duchenne muscular
dystrophy (DMD) in patients who have a confirmed mutation of the DMD
gene that is amenable to exon 53 skipping.
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 VYONDYS (golodirsen), go to the ``Drugs@FDA'' website
at https://www.accessdata.fda.gov/scripts/cder/daf/.
Dated: January 16, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-01059 Filed 1-22-20; 8:45 am]
BILLING CODE 4164-01-P