[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
``Drugs@FDA'' 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]
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