Issuance of Priority Review Voucher; Rare Pediatric Disease Product, 64133 [2018-27043]
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Federal Register / Vol. 83, No. 239 / Thursday, December 13, 2018 / Notices
FOR FURTHER INFORMATION CONTACT:
amozie on DSK3GDR082PROD with NOTICES1
Karen Takahashi, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave. Bldg. 75, Rm. 6686,
Silver Spring, MD 20993–0002, 301–
796–3191; Stephen Ripley, 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; or Jonathan Bray, Center
for Veterinary Medicine (HFV–232),
Food and Drug Administration, 7519
Standish Pl., Rm. 130, Rockville, MD
20855, 240–402–5623.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a guidance for industry entitled ‘‘Data
Integrity and Compliance With Drug
CGMP: Questions and Answers.’’ In
recent years, FDA has increasingly
observed CGMP violations involving
data integrity during CGMP inspections.
This is troubling because ensuring data
integrity is an important component of
industry’s responsibility to ensure the
safety, efficacy, and quality of drugs,
and of FDA’s ability to protect the
public health. These data integrityrelated CGMP violations have led to
numerous regulatory actions, including
warning letters, import alerts, and
consent decrees. The underlying
premise in 21 CFR 210.1 and 212.2 is
that CGMP sets forth minimum
requirements to assure that drugs meet
the standards of the Federal Food, Drug,
and Cosmetic Act regarding safety,
identity, strength, quality, and purity.
The guidance addresses specific
questions about how data integrity
relates to compliance with CGMP for
drugs, as well as more general data
integrity concepts, in question and
answer format. This guidance was
published as a draft guidance in April
2016—‘‘Data Integrity and Compliance
With CGMP’’—and has been revised in
response to comments from the docket
for clarity. Other comments to the
docket requested additional details on
FDA’s thinking on current best practices
and additional examples. The Agency
has used clarifying language and
additional examples that also address
best practices for ensuring data
integrity. A paragraph regarding
independent security role assignments
for small operations or facilities was
removed because the guidance for
industry ‘‘PET Drugs—Current Good
Manufacturing Practice (CGMP)’’
covering this topic is sufficiently clear.
This guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
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17:12 Dec 12, 2018
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The guidance represents the current
thinking of FDA on data integrity and
compliance with drug CGMP. 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 requirements of the
applicable statutes and regulations. This
guidance is not subject to Executive
Order 12866.
II. Paperwork Reduction Act of 1995
This guidance refers to previously
approved collections of information that
are subject to review by the Office of
Management and Budget (OMB) under
the Paperwork Reduction Act of 1995
(44 U.S.C. 3501–3520). The collections
of information in 21 CFR parts 210 and
211 (CGMPs), 212 (positron emission
tomography CGMPs), and 11 (electronic
records and signatures) have been
approved under OMB control numbers
0910–0139, 0910–0667, and 0910–0303,
respectively.
III. Electronic Access
Persons with access to the internet
may obtain the guidance at either
https://www.fda.gov/Drugs/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm, https://
www.fda.gov/BiologicsBloodVaccines/
GuidanceComplianceRegulatory
Information/default.htm, https://
www.fda.gov/AnimalVeterinary/
GuidanceComplianceEnforcement/
GuidanceforIndustry/default.htm, or
https://www.regulations.gov.
Dated: December 7, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–26957 Filed 12–12–18; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2018–N–4609]
Issuance of Priority Review Voucher;
Rare Pediatric Disease Product
AGENCY:
Food and Drug Administration,
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), as
amended by the Food and Drug
Administration Safety and Innovation
Act (FDASIA), authorizes FDA to award
priority review vouchers to sponsors of
SUMMARY:
PO 00000
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64133
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 GAMIFANT
(emapalumab-lzsg) Injection,
manufactured by Novimmune S.A.,
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: althea.cuff@fda.hhs.gov.
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 GAMIFANT
(emapalumab-lzsg) Injection,
manufactured by Novimmune S.A.,
meets the criteria for a priority review
voucher. GAMIFANT (emapalumablzsg) Injection is indicated for the
treatment of adult and pediatric
(newborn and older) patients with
primary hemophagocytic
lymphohistiocytosis (HLH) with
refractory, recurrent or progressive
disease or intolerance with conventional
HLH therapy.
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/
DevelopingProductsforRareDiseases
Conditions/RarePediatricDiseasePriority
VoucherProgram/default.htm. For
further information about GAMIFANT
(emapalumab-lzsg) Injection, go to the
‘‘Drugs@FDA’’ website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
SUPPLEMENTARY INFORMATION:
Dated: December 10, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–27043 Filed 12–12–18; 8:45 am]
BILLING CODE 4164–01–P
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[Federal Register Volume 83, Number 239 (Thursday, December 13, 2018)]
[Notices]
[Page 64133]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-27043]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-N-4609]
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 GAMIFANT (emapalumab-lzsg) Injection, manufactured by Novimmune
S.A., 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
GAMIFANT (emapalumab-lzsg) Injection, manufactured by Novimmune S.A.,
meets the criteria for a priority review voucher. GAMIFANT (emapalumab-
lzsg) Injection is indicated for the treatment of adult and pediatric
(newborn and older) patients with primary hemophagocytic
lymphohistiocytosis (HLH) with refractory, recurrent or progressive
disease or intolerance with conventional HLH therapy.
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 GAMIFANT (emapalumab-lzsg) Injection, go to the
``[email protected]'' website at https://www.accessdata.fda.gov/scripts/cder/daf/.
Dated: December 10, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-27043 Filed 12-12-18; 8:45 am]
BILLING CODE 4164-01-P
