[Federal Register Volume 84, Number 121 (Monday, June 24, 2019)]
[Notices]
[Pages 29520-29521]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-13356]


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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 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 ZOLGENSMA (onasemnogene abeparvovec-xioi),

[[Page 29521]]

manufactured by AveXis, Inc., meets the criteria for a priority review 
voucher.

FOR FURTHER INFORMATION CONTACT: Shruti Modi, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.

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 upon approval of those 
applications. FDA has determined that ZOLGENSMA (onasemnogene 
abeparvovec-xioi), manufactured by AveXis, Inc., meets the criteria for 
a priority review voucher. ZOLGENSMA (onasemnogene abeparvovec-xioi) is 
indicated for the treatment of pediatric patients less than 2 years of 
age with spinal muscular atrophy with biallelic mutations in the 
survival motor neuron 1 gene.
    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 ZOLGENSMA (onasemnogene abeparvovec-xioi), go to the 
Center for Biologics Evaluation and Research Cellular and Gene Therapy 
Products website at https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.

    Dated: June 18, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-13356 Filed 6-21-19; 8:45 am]
BILLING CODE 4164-01-P


