83 FR 64133 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 83, Issue 239 (December 13, 2018)
Food and Drug Administration
Federal Register Volume 83, Issue 239 (December 13, 2018)
| Page Range | 64133-64133 | |
| FR Document | 2018-27043 |
[Federal Register Volume 83, Number 239 (Thursday, December 13, 2018)] [Notices] [Page 64133] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2018-27043] ----------------------------------------------------------------------- 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
![Federal Register / Vol. 83, No. 239 / Thursday, December 13, 2018 / Notices 64133
FOR FURTHER INFORMATION CONTACT: The guidance represents the current approved rare pediatric disease product
Karen Takahashi, Center for Drug thinking of FDA on data integrity and applications that meet certain criteria.
Evaluation and Research, Food and compliance with drug CGMP. It does FDA is required to publish notice of the
Drug Administration, 10903 New not establish any rights for any person award of the priority review voucher.
Hampshire Ave. Bldg. 75, Rm. 6686, and is not binding on FDA or the public. FDA has determined that GAMIFANT
Silver Spring, MD 20993–0002, 301– You can use an alternative approach if (emapalumab-lzsg) Injection,
796–3191; Stephen Ripley, Center for it satisfies the requirements of the manufactured by Novimmune S.A.,
Biologics Evaluation and Research, applicable statutes and regulations. This meets the criteria for a priority review
Food and Drug Administration, 10903 guidance is not subject to Executive voucher.
New Hampshire Ave. Bldg. 71, Rm. Order 12866.
7301, Silver Spring, MD 20993–0002, FOR FURTHER INFORMATION CONTACT:
II. Paperwork Reduction Act of 1995
240–402–7911; or Jonathan Bray, Center Althea Cuff, Center for Drug Evaluation
for Veterinary Medicine (HFV–232), This guidance refers to previously and Research, Food and Drug
Food and Drug Administration, 7519 approved collections of information that Administration, 10903 New Hampshire
Standish Pl., Rm. 130, Rockville, MD are subject to review by the Office of Ave., Silver Spring, MD 20993–0002,
20855, 240–402–5623. Management and Budget (OMB) under 301–796–4061, Fax: 301–796–9856,
SUPPLEMENTARY INFORMATION: the Paperwork Reduction Act of 1995 email: althea.cuff@fda.hhs.gov.
(44 U.S.C. 3501–3520). The collections
I. Background of information in 21 CFR parts 210 and SUPPLEMENTARY INFORMATION: FDA is
FDA is announcing the availability of 211 (CGMPs), 212 (positron emission announcing the issuance of a priority
a guidance for industry entitled ‘‘Data tomography CGMPs), and 11 (electronic review voucher to the sponsor of an
Integrity and Compliance With Drug records and signatures) have been approved rare pediatric disease product
CGMP: Questions and Answers.’’ In approved under OMB control numbers application. Under section 529 of the
recent years, FDA has increasingly 0910–0139, 0910–0667, and 0910–0303, FD&C Act (21 U.S.C. 360ff), which was
observed CGMP violations involving respectively. added by FDASIA, FDA will award
data integrity during CGMP inspections. priority review vouchers to sponsors of
III. Electronic Access
This is troubling because ensuring data approved rare pediatric disease product
integrity is an important component of Persons with access to the internet
applications that meet certain criteria.
industry’s responsibility to ensure the may obtain the guidance at either
FDA has determined that GAMIFANT
safety, efficacy, and quality of drugs, https://www.fda.gov/Drugs/Guidance
(emapalumab-lzsg) Injection,
and of FDA’s ability to protect the ComplianceRegulatoryInformation/
Guidances/default.htm, https:// manufactured by Novimmune S.A.,
public health. These data integrity-
www.fda.gov/BiologicsBloodVaccines/ meets the criteria for a priority review
related CGMP violations have led to
GuidanceComplianceRegulatory voucher. GAMIFANT (emapalumab-
numerous regulatory actions, including
warning letters, import alerts, and Information/default.htm, https:// lzsg) Injection is indicated for the
consent decrees. The underlying www.fda.gov/AnimalVeterinary/ treatment of adult and pediatric
premise in 21 CFR 210.1 and 212.2 is GuidanceComplianceEnforcement/ (newborn and older) patients with
that CGMP sets forth minimum GuidanceforIndustry/default.htm, or primary hemophagocytic
requirements to assure that drugs meet https://www.regulations.gov. lymphohistiocytosis (HLH) with
the standards of the Federal Food, Drug, Dated: December 7, 2018. refractory, recurrent or progressive
and Cosmetic Act regarding safety, Leslie Kux,
disease or intolerance with conventional
identity, strength, quality, and purity. HLH therapy.
Associate Commissioner for Policy.
The guidance addresses specific For further information about the Rare
[FR Doc. 2018–26957 Filed 12–12–18; 8:45 am]
questions about how data integrity Pediatric Disease Priority Review
BILLING CODE 4164–01–P
relates to compliance with CGMP for Voucher Program and for a link to the
drugs, as well as more general data
full text of section 529 of the FD&C Act,
integrity concepts, in question and
DEPARTMENT OF HEALTH AND go to https://www.fda.gov/ForIndustry/
answer format. This guidance was
HUMAN SERVICES DevelopingProductsforRareDiseases
published as a draft guidance in April
2016—‘‘Data Integrity and Compliance Conditions/RarePediatricDiseasePriority
Food and Drug Administration VoucherProgram/default.htm. For
With CGMP’’—and has been revised in
response to comments from the docket [Docket No. FDA–2018–N–4609] further information about GAMIFANT
for clarity. Other comments to the (emapalumab-lzsg) Injection, go to the
docket requested additional details on Issuance of Priority Review Voucher; ‘‘Drugs@FDA’’ website at https://
FDA’s thinking on current best practices Rare Pediatric Disease Product www.accessdata.fda.gov/scripts/cder/
and additional examples. The Agency AGENCY: Food and Drug Administration, daf/.
has used clarifying language and HHS. Dated: December 10, 2018.
additional examples that also address ACTION: Notice. Leslie Kux,
best practices for ensuring data
integrity. A paragraph regarding SUMMARY: The Food and Drug Associate Commissioner for Policy.
independent security role assignments Administration (FDA) is announcing the [FR Doc. 2018–27043 Filed 12–12–18; 8:45 am]
for small operations or facilities was issuance of a priority review voucher to BILLING CODE 4164–01–P
amozie on DSK3GDR082PROD with NOTICES1
removed because the guidance for the sponsor of a rare pediatric disease
industry ‘‘PET Drugs—Current Good product application. The Federal Food,
Manufacturing Practice (CGMP)’’ Drug, and Cosmetic Act (FD&C Act), as
covering this topic is sufficiently clear. amended by the Food and Drug
This guidance is being issued Administration Safety and Innovation
consistent with FDA’s good guidance Act (FDASIA), authorizes FDA to award
practices regulation (21 CFR 10.115). priority review vouchers to sponsors of
VerDate Sep<11>2014 17:12 Dec 12, 2018 Jkt 247001 PO 00000 Frm 00037 Fmt 4703 Sfmt 9990 E:\FR\FM\13DEN1.SGM 13DEN1](https://thefederalregister.org/doc/83_FR_64372/page/1.svg)
Document Created: 2018-12-13 01:15:20
Document Modified: 2018-12-13 01:15:20
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| 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] | |
| FR Citation | 83 FR 64133 |
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