Issuance of Priority Review Voucher; Rare Pediatric Disease Product, 35307 [2021-14191]
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Federal Register / Vol. 86, No. 125 / Friday, July 2, 2021 / Notices
trials conducted exclusively in patients
with CNS metastases.
CNS metastases are associated with
significant morbidity and mortality and
development of therapeutic products for
patients with CNS metastases is needed.
FDA has participated in efforts to
facilitate drug development for patients
with CNS metastases, including a March
2019 ‘‘Workshop on Product
Development for CNS Metastases.’’
Stakeholders at this meeting stated there
is a need for further FDA guidance on
specific topics, including identifying
optimal study endpoints. Study design
challenges for CNS metastases include
uncertainty regarding optimal
endpoints, lack of standardized
response assessments, understanding
how CNS metastases are evaluated in
the context of the entire burden of
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drug’s potential benefit (e.g., timing of
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to other sites of metastases), and
interpreting radiographic response in
the setting of recent radiation therapy or
surgery. This guidance is intended to
provide recommendations on these
study design challenges.
In the Federal Register of August 27,
2020 (85 FR 53007), FDA announced the
availability of the draft guidance
‘‘Evaluating Cancer Drugs in Patients
with Central Nervous System
Metastases’’ dated August 2020. FDA
received several comments on the draft
guidance and those comments were
considered as the guidance was
finalized. A summary of changes
includes: Clarification on the number of
stratification factors the protocol should
specify in order to minimize bias,
confirmation of the version of Response
Evaluation Criteria in Solid Tumours
(RECIST) that should be referred to
when evaluating CNS disease,
clarification that both CNS and
systematic duration of response should
be captured and the addition of a 6month timepoint, and the addition of
progression-free survival in patients
with brain metastasis as another
measurement to be reported when CNS
is a common metastatic site. In addition,
editorial changes were made to improve
clarity. The guidance announced in this
notice finalizes the draft guidance dated
August 27, 2020.
This guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
The guidance represents the current
thinking of FDA on ‘‘Evaluating Cancer
Drugs in Patients with Central Nervous
System Metastases.’’ It does not
establish any rights for any person and
is not binding on FDA or the public.
You can use an alternative approach if
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Persons with access to the internet
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biologics-guidances or https://
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Dated: June 28, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for
Policy.
[FR Doc. 2021–14194 Filed 7–1–21; 8:45 am]
BILLING CODE 4164–01–P
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,
35307
(plasminogen, human-tvmh),
manufactured by Prometic
Bioproduction, 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 RYPLAZIM (plasminogen, humantvmh), manufactured by Prometic
Bioproduction, Inc., meets the criteria
for a priority review voucher.
RYPLAZIM (plasminogen, human-tvmh)
is indicated for the treatment of patients
with plasminogen deficiency type 1
(hypoplasminogenemia).
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 RYPLAZIM
(plasminogen, human-tvmh), 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-therapyproducts.
Dated: June 25, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for
Policy.
[FR Doc. 2021–14191 Filed 7–1–21; 8:45 am]
BILLING CODE 4164–01–P
HHS.
ACTION:
Notice.
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 RYPLAZIM
SUMMARY:
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The meeting will be closed to the
public in accordance with the
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[Federal Register Volume 86, Number 125 (Friday, July 2, 2021)]
[Notices]
[Page 35307]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-14191]
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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.
-----------------------------------------------------------------------
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 RYPLAZIM (plasminogen, human-tvmh), manufactured by Prometic
Bioproduction, 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 RYPLAZIM (plasminogen, human-tvmh),
manufactured by Prometic Bioproduction, Inc., meets the criteria for a
priority review voucher. RYPLAZIM (plasminogen, human-tvmh) is
indicated for the treatment of patients with plasminogen deficiency
type 1 (hypoplasminogenemia).
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
RYPLAZIM (plasminogen, human-tvmh), 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 25, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-14191 Filed 7-1-21; 8:45 am]
BILLING CODE 4164-01-P
