Issuance of Priority Review Voucher; Rare Pediatric Disease Product, 1925 [2024-00401]
Download as PDF
<![CDATA[
Federal Register / Vol. 89, No. 8 / Thursday, January 11, 2024 / Notices
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 FILSUVEZ (birch
triterpenes), manufactured by Amryt
Pharmaceuticals, meets the criteria for a
priority review voucher.
FOR FURTHER INFORMATION CONTACT:
Cathryn Lee, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–1394.
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 FILSUVEZ (birch triterpenes),
manufactured by Amryt
Pharmaceuticals, meets the criteria for a
priority review voucher. FILSUVEZ
(birch triterpenes) gel is indicated for
the treatment of wounds associated with
dystrophic and junctional epidermolysis
bullosa in adult and pediatric patients 6
months of age and older.
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/
DevelopingProductsforRare
DiseasesConditions/RarePediatric
DiseasePriorityVoucherProgram/
default.htm. For further information
about FILSUVEZ (birch triterpenes), go
to the ‘‘Drugs@FDA’’ website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
Dated: January 8, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024–00400 Filed 1–10–24; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
ddrumheller on DSK120RN23PROD with NOTICES1
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:
17:31 Jan 10, 2024
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.
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 ADZYNMA (ADAMTS13,
recombinant-krhn), manufactured by
Takeda Pharmaceuticals U.S.A., Inc.,
meets the criteria for a priority review
voucher.
ADZYNMA (ADAMTS13,
recombinant-krhn) is indicated for
prophylactic or on-demand enzyme
replacement therapy in adult and
pediatric patients with congenital
thrombotic thrombocytopenic purpura.
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/industry/
developing-products-rare-diseasesconditions/rare-pediatric-disease-rpddesignation-and-voucher-programs. For
further information about ADZYNMA
(ADAMTS13, recombinant-krhn), go to
the Center for Biologics Evaluation and
Research’s Approved Blood Products
website at https://www.fda.gov/
vaccines-blood-biologics/adzynma.
SUPPLEMENTARY INFORMATION:
Dated: January 8, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024–00401 Filed 1–10–24; 8:45 am]
Notice.
VerDate Sep<11>2014
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)
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 ADZYNMA
(ADAMTS13, recombinant-krhn),
manufactured by Takeda
Pharmaceuticals U.S.A., Inc., meets the
criteria for a priority review voucher.
SUMMARY:
BILLING CODE 4164–01–P
Jkt 262001
PO 00000
Frm 00051
Fmt 4703
Sfmt 4703
1925
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2022–D–2512]
Q5A(R2) Viral Safety Evaluation of
Biotechnology Products Derived From
Cell Lines of Human or Animal Origin;
International Council for
Harmonisation; Guidance for Industry;
Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance for industry entitled ‘‘Q5A(R2)
Viral Safety Evaluation of Biotechnology
Products Derived from Cell Lines of
Human or Animal Origin.’’ The
guidance was prepared under the
auspices of the International Council for
Harmonisation of Technical
Requirements for Pharmaceuticals for
Human Use (ICH). The guidance is
intended to describe risk-based
principles and mitigation strategies to
assure the viral safety of biotechnology
products, including the data necessary
to submit in a marketing application.
The guidance also finalizes the updates
based on advances in scientific
knowledge and regulatory expectations
to the first version of the ICH guidance
for industry ‘‘Q5A Viral Safety
Evaluation of Biotechnology Products
Derived from Cell Lines of Human or
Animal Origin,’’ issued in September
1998. Lastly, the guidance replaces the
draft guidance ‘‘Q5A(R2) Viral Safety
Evaluation of Biotechnology Products
Derived from Cell Lines of Human or
Animal Origin’’ issued on November 14,
2022.
DATES: The announcement of the
guidance is published in the Federal
Register on January 11, 2024.
ADDRESSES: You may submit either
electronic or written comments on
Agency guidances at any time as
follows:
SUMMARY:
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
instructions for submitting comments.
Comments submitted electronically,
including attachments, to https://
www.regulations.gov will be posted to
the docket unchanged. Because your
comment will be made public, you are
solely responsible for ensuring that your
comment does not include any
E:\FR\FM\11JAN1.SGM
11JAN1
]]>Agencies
[Federal Register Volume 89, Number 8 (Thursday, January 11, 2024)]
[Notices]
[Page 1925]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-00401]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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) 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 ADZYNMA (ADAMTS13, recombinant-krhn), manufactured by Takeda
Pharmaceuticals U.S.A., 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 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 ADZYNMA (ADAMTS13, recombinant-krhn),
manufactured by Takeda Pharmaceuticals U.S.A., Inc., meets the criteria
for a priority review voucher.
ADZYNMA (ADAMTS13, recombinant-krhn) is indicated for prophylactic
or on-demand enzyme replacement therapy in adult and pediatric patients
with congenital thrombotic thrombocytopenic purpura.
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/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs. For further information about ADZYNMA
(ADAMTS13, recombinant-krhn), go to the Center for Biologics Evaluation
and Research's Approved Blood Products website at https://www.fda.gov/vaccines-blood-biologics/adzynma.
Dated: January 8, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-00401 Filed 1-10-24; 8:45 am]
BILLING CODE 4164-01-P
