80 FR 58489 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 188 (September 29, 2015)

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 rare pediatric disease product applications that meet certain criteria. FDA has determined that Xuriden (uridine triacetate), manufactured by Wellstat Therapeutics Corp., meets the criteria for a priority review voucher.

[Federal Register Volume 80, Number 188 (Tuesday, September 29, 2015)]
[Notices]
[Page 58489]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-24640]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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 rare pediatric disease product 
applications that meet certain criteria. FDA has determined that 
Xuriden (uridine triacetate), manufactured by Wellstat Therapeutics 
Corp., 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 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 Xuriden (uridine triacetate), 
manufactured by Wellstat Therapeutics Corp., meets the criteria for a 
priority review voucher. Uridine triacetate is a pyrimidine analog for 
uridine replacement. Xuriden is indicated for the treatment of 
hereditary orotic aciduria. Hereditary orotic aciduria is caused by a 
deficiency in the activity of the pyrimidine pathway enzyme uridine 5'-
monophosphate synthase. The disorder is generally characterized by 
anemia and/or other hematological manifestations, excessive urinary 
excretion of orotic acid, failure to thrive, and developmental delay.
    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 Xuriden (uridine triacetate), go to 
the Drugs@FDA Web site at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm.

    Dated: September 24, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-24640 Filed 9-28-15; 8:45 am]
 BILLING CODE 4164-01-P