Issuance of Priority Review Voucher; Rare Pediatric Disease Product
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Issuing agencies
Abstract
The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the applicant of a rare pediatric disease 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 SKYSONA (elivaldogene autotemcel), manufactured by bluebird bio, Inc., meets the criteria for a priority review voucher.
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<title>Federal Register, Volume 87 Issue 194 (Friday, October 7, 2022)</title>
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[Federal Register Volume 87, Number 194 (Friday, October 7, 2022)]
[Notices]
[Pages 61090-61091]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-21851]
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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 applicant of a rare
pediatric disease product application. The Federal Food, Drug, and
Cosmetic Act (FD&C Act) authorizes FDA to award priority review
[[Page 61091]]
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 SKYSONA (elivaldogene autotemcel), manufactured by bluebird bio,
Inc., meets the criteria for a priority review voucher.
FOR FURTHER INFORMATION CONTACT: Myrna Hanna, 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 applicant 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 SKYSONA (elivaldogene autotemcel), manufactured
by bluebird bio, Inc., meets the criteria for a priority review
voucher. SKYSONA is indicated to slow the progression of neurologic
dysfunction in boys 4 to 17 years of age with early active cerebral
adrenoleukodystrophy.
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 <a href="https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs">https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs</a>. For further information about
SKYSONA, go to the Center for Biologics Evaluation and Research
Cellular and Gene Therapy Products website at <a href="https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products">https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products</a>.
Dated: September 30, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-21851 Filed 10-6-22; 8:45 am]
BILLING CODE 4164-01-P
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