[Federal Register Volume 83, Number 1 (Tuesday, January 2, 2018)]
[Notices]
[Page 125]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-28256]


=======================================================================
-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0809]


Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

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 LUXTURNA (voretigene neparvovec), manufactured by Spark 
Therapeutics, Inc., meets the criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Gretchen Opper, 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 LUXTURNA (voretigene neparvovec), 
manufactured by Spark Therapeutics, Inc., meets the criteria for a 
priority review voucher. LUXTURNA (voretigene neparvovec) is an adeno-
associated virus vector-based gene therapy indicated for the treatment 
of patients with confirmed biallelic RPE65 mutation-associated retinal 
dystrophy. Patients must have viable retinal cells as determined by the 
treating physician(s).
    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 LUXTURNA (voretigene neparvovec), go to the Center 
for Biologics Evaluation and Research cellular and gene therapy 
products website at https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/default.htm.

    Dated: December 26, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-28256 Filed 12-29-17; 8:45 am]
BILLING CODE 4164-01-P