Document ID: FDA-2018-N-4609-0002
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2019-03-29T04:00Z

[Federal Register Volume 84, Number 61 (Friday, March 29, 2019)]
[Notices]
[Pages 11985-11986]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-06138]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 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

[[Page 11986]]

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 SYMDEKO 
(tezacaftor/ivacaftor), manufactured by Vertex Pharmaceutical, 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 protected].

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 
SYMDEKO (tezacaftor/ivacaftor), manufactured by Vertex Pharmaceutical, 
Inc., meets the criteria for a priority review voucher. SYMDEKO 
(tezacaftor/ivacaftor) is indicated for the treatment of patients with 
cystic fibrosis aged 12 years and older who are homozygous for the 
F508del mutation or who have at least one mutation in the cystic 
fibrosis transmembrane conductance regulator gene that is responsive to 
tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
    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 SYMDEKO (tezacaftor/ivacaftor), go to the 
``[email protected]'' website at https://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: March 26, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-06138 Filed 3-28-19; 8:45 am]
 BILLING CODE 4164-01-P