Document ID: FDA-2020-N-0026-0007
Agency: fda
Document Type: Notice
Title: Issuance of Priority Review Voucher; Rare Pediatric Disease Product
Posted Date: 2020-12-17T05:00Z

[Federal Register Volume 85, Number 243 (Thursday, December 17, 2020)]
[Notices]
[Pages 81933-81934]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-27778]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-N-0026]

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

[[Page 81934]]

product application. The Federal Food, Drug, and Cosmetic Act (FD&C 
Act) 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 ZOKINVY (lonafarnib), 
manufactured by Eiger BioPharmaceuticals, 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), FDA will award priority review vouchers to sponsors of approved 
rare pediatric disease product applications that meet certain criteria. 
FDA has determined that ZOKINVY (lonafarnib), manufactured by Eiger 
BioPharmaceuticals, Inc., meets the criteria for a priority review 
voucher.
    ZOKINVY (lonafarnib) is indicated in patients 12 months of age and 
older with a body surface area of 0.39 m\2\ and above:

 To reduce the risk of mortality in Hutchinson-Gilford progeria 
syndrome
 For the treatment of processing-deficient progeroid 
laminopathies with either:
    [cir] Heterozygous LMNA mutation with progerin-like protein 
accumulation or
    [cir] Homozygous or compound heterozygous ZMPSTE24 mutations

    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 ZOKINVY (lonafarnib), go to the ``[email protected]'' website 
at http://www.accessdata.fda.gov/scripts/cder/daf/.

    Dated: December 14, 2020.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2020-27778 Filed 12-16-20; 8:45 am]
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