Document ID: FDA-2014-N-0229-0008
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2016-10-17T04:00Z

[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: larry.bauer@fda.hhs.gov.

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]
 BILLING CODE 4164-01-P