Issuance of Priority Review Voucher; Rare Pediatric Disease Product, 55304 [2020-19604]
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Federal Register / Vol. 85, No. 173 / Friday, September 4, 2020 / Notices
the liver, and glomerular filtration and
tubular secretion of unchanged drug by
the kidneys (i.e., renal excretion). If a
drug is eliminated primarily through
renal excretion, then impaired renal
function usually alters the drug’s PK to
an extent that the dosage regimen may
need to be changed from that used in
patients with normal renal function. For
most drugs that are likely to be
administered to patients with impaired
renal function, it is important to
characterize PK in subjects with
impaired renal function to provide
appropriate dosing recommendations.
The safety and efficacy of a drug are
generally established for a particular
dosage regimen (or range of dosage
regimens) in late-phase clinical trials
that enroll patients from the target
patient population. Frequently,
however, individuals with advanced
kidney disease are explicitly excluded
from participation in these studies,
hindering the assessment of the effects
of severely impaired kidney function on
the PK of a drug or the patient’s clinical
response. A well-planned drug
development program can enable
prospective dosage adjustment based on
the observed or expected changes in the
PK of a drug due to impaired renal
function prior to initiating phase 2 or
phase 3 trials.
This guidance replaces the 2010
version and provides updated
recommendations on the following
topics:
(1) When a dedicated study of a drug’s
PK in subjects with impaired renal
function is recommended and when it
may not be needed;
(2) The design and conduct of
pharmacokinetic studies in subjects
with impaired renal function;
(3) Considerations for characterizing a
drug’s PK in patients undergoing
intermittent or continuous dialytic
therapies;
(4) The use of pharmacokinetic
information from phase 2 and 3 studies
to inform dosing recommendations for
patients with renal impairment; and
(5) The analysis and reporting of the
results of studies that characterize the
impact of renal impairment and how
these data inform dosing.
This draft guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
The draft guidance, when finalized, will
represent the current thinking of FDA
on ‘‘Pharmacokinetics in Patients with
Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing.’’ 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 it satisfies the
VerDate Sep<11>2014
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requirements of the applicable statutes
and regulations.
II. Paperwork Reduction Act of 1995
FDA tentatively concludes that this
draft guidance contains no collection of
information. Therefore, clearance by the
Office of Management and Budget
(OMB) under the Paperwork Reduction
Act of 1995 (PRA) (44 U.S.C. 3501–
3521) is not required.
However, this draft guidance refers to
previously approved FDA collections of
information. These collections of
information are subject to review by
OMB under the PRA. The collection of
information in 21 CFR 201.57 has been
approved under OMB control number
0910–0572.
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs or https://
www.regulations.gov.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–19597 Filed 9–3–20; 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
Food and Drug Administration,
Health and Human Services (HHS).
ACTION: Notice.
AGENCY:
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 (the 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 VILTEPSO
(viltolarsen) manufactured by Nippon
Shinyaku Co., Ltd. (NS Pharma Inc.,
U.S. Agent), meets the criteria for a
priority review voucher.
FOR FURTHER INFORMATION CONTACT:
Althea Cuff, Center for Drug Evaluation
SUMMARY:
PO 00000
Frm 00053
Fmt 4703
Sfmt 4703
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–4061, Fax: 301–796–9856,
email: althea.cuff@fda.hhs.gov.
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.
FDA has determined that VILTEPSO
(viltolarsen) manufactured by Nippon
Shinyaku Co., Ltd. (NS Pharma Inc.,
U.S. Agent), meets the criteria for a
priority review voucher.
VILTEPSO (viltolarsen) is indicated
for the treatment of Duchenne Muscular
Dystrophy in patients amenable to Exon
53 Skipping.
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/
DevelopingProductsfor
RareDiseasesConditions/
RarePediatricDiseasePriorityVoucher
Program/default.htm. FOR FURTHER
INFORMATION about VILTEPSO
(viltolarsen) go to the ‘‘Drugs@FDA’’
website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–19604 Filed 9–3–20; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Health Resources and Services
Administration
Charter Renewal for the Advisory
Committee on Organ Transplantation
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services (HHS).
ACTION: Notice.
AGENCY:
In accordance with the
Federal Advisory Committee Act
(FACA), HHS is hereby giving notice
that the Advisory Committee on Organ
Transplantation (ACOT) has been
renewed. The effective date of the
renewed charter is August 31, 2020.
FOR FURTHER INFORMATION CONTACT:
Robert Walsh, Designated Federal
Officer, HRSA Division of
SUMMARY:
E:\FR\FM\04SEN1.SGM
04SEN1
]]>Agencies
[Federal Register Volume 85, Number 173 (Friday, September 4, 2020)]
[Notices]
[Page 55304]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-19604]
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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, Health and Human Services (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 (the 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 VILTEPSO (viltolarsen) manufactured by Nippon
Shinyaku Co., Ltd. (NS Pharma Inc., U.S. Agent), meets the criteria for
a priority review voucher.
FOR FURTHER INFORMATION CONTACT: Althea Cuff, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: 301-
796-9856, email: [email protected].
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. FDA has determined that
VILTEPSO (viltolarsen) manufactured by Nippon Shinyaku Co., Ltd. (NS
Pharma Inc., U.S. Agent), meets the criteria for a priority review
voucher.
VILTEPSO (viltolarsen) is indicated for the treatment of Duchenne
Muscular Dystrophy in patients amenable to Exon 53 Skipping.
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 VILTEPSO (viltolarsen) go to the ``[email protected]''
website at https://www.accessdata.fda.gov/scripts/cder/daf/.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-19604 Filed 9-3-20; 8:45 am]
BILLING CODE 4164-01-P
