[Federal Register Volume 81, Number 200 (Monday, October 17, 2016)]
[Notices]
[Page 71511]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-24947]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2014-N-0229]
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
Administration Safety and Innovation Act (FDASIA), authorizes FDA to
award priority review vouchers to sponsors of rare pediatric disease
product applications that meet certain criteria. FDA has determined
that EXONDYS 51 (eteplirsen), manufactured by Sarepta Therapeutics,
meets the criteria for a priority review voucher.
FOR FURTHER INFORMATION CONTACT: Larry Bauer, Rare Diseases Program,
Center for Drug Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4842,
FAX: 301-796-9858, email: [email protected].
SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority
review voucher to the sponsor of a 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 rare pediatric disease product applications that meet
certain criteria. FDA has determined that EXONDYS 51 (eteplirsen),
manufactured by Sarepta Therapeutics, meets the criteria for a priority
review voucher. EXONDYS 51 (eteplirsen) 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 51 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 http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further
information about EXONDYS 51 (eteplirsen) go to the ``Drugs@FDA'' Web
site at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm.
Dated: October 6, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-24947 Filed 10-14-16; 8:45 am]
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