Document ID: FDA-2018-N-4609-0004
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2019-07-01T04:00Z

[Federal Register Volume 84, Number 126 (Monday, July 1, 2019)]
[Notices]
[Page 31324]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-13944]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket Nos. FDA-2017-N-0809 and FDA-2018-N-4609]

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 (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 approved rare pediatric disease product 
applications that meet certain criteria. FDA is required to publish 
notice of the award of the priority review voucher. FDA has determined 
that KANUMA (sebelipase alfa), manufactured by Alexion Pharmaceuticals 
Inc., meets the criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Althea Cuff, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301-
796-9856, email: althea.cuff@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority 
review voucher to the sponsor of an approved 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 approved rare pediatric disease product 
applications that meet certain criteria. FDA has determined that KANUMA 
(sebelipase alfa), manufactured by Alexion Pharmaceuticals Inc., meets 
the criteria for a priority review voucher. KANUMA (sebelipase alfa), 
is indicated for the treatment of patients with a diagnosis of 
Lysosomal Acid Lipase deficiency.
    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 https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further 
information about KANUMA (sebelipase alfa), go to the ``[email protected]'' 
website at https://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: June 25, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-13944 Filed 6-28-19; 8:45 am]
 BILLING CODE 4164-01-P