Document ID: FDA-2014-N-0229-0001
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2014-03-12T04:00Z

[Federal Register Volume 79, Number 48 (Wednesday, March 12, 2014)]
[Notices]
[Pages 14055-14056]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-05410]

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

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 VIMIZIM (elosulfase alfa), manufactured by BioMarin 
Pharmaceutical, Inc., meets the criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Vicki Moyer, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6467, Silver Spring, MD 20993-0002, 301-
796-2200, FAX: 301-796-9855, vicki.moyer@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 VIMIZIM (elosulfase alfa), manufactured by BioMarin 
Pharmaceutical, Inc., meets the criteria for a priority review

[[Page 14056]]

voucher. VIMIZIM (elosulfase alfa) is indicated for the treatment of 
Mucopolysaccharidosis Type IV A (Morquio A syndrome). Morquio A 
syndrome is a rare congenital disorder caused by the absence or 
malfunctioning of an enzyme involved in an important metabolic pathway, 
leading to problems with bone development, growth, and movement.
    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 VIMIZIM (elosulfase alfa), go to the 
Drugs@FDA Web site at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm.

    Dated: March 7, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-05410 Filed 3-11-14; 8:45 am]
BILLING CODE 4160-01-P