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