Document ID: FDA-2014-N-0229-0003
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2015-04-07T04:00Z

[Federal Register Volume 80, Number 66 (Tuesday, April 7, 2015)]
[Notices]
[Page 18628]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2015-08016]

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

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 (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 CHOLBAM (cholic acid), manufactured by Asklepion Pharmaceuticals, 
LLC, 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), 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 CHOLBAM (cholic acid), manufactured by Asklepion 
Pharmaceuticals, LLC, meets the criteria for a priority review voucher. 
CHOLBAM (cholic acid) is a bile acid indicated for the treatment of 
bile acid synthesis disorders due to single enzyme defects and as 
adjunctive treatment of peroxisomal disorders, including Zellweger 
spectrum disorders in patients who exhibit manifestations of liver 
disease or steatorrhea or complications from decreased fat soluble 
vitamin absorption. Bile acid synthesis disorders is a group of rare 
congenital disorders caused by the absence or malfunction of an enzyme 
involved in an important metabolic pathway, leading to a failure to 
produce normal bile acids.
    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 CHOLBAM (cholic acid), go to the 
Drugs@FDA Web site at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm.

    Dated: April 2, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-08016 Filed 4-6-15; 8:45 am]
BILLING CODE CODE 4164-01-P