82 FR 42686 - Issuance of Priority Review Voucher; Rare Pediatric Disease Product
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 82, Issue 174 (September 11, 2017)
Food and Drug Administration
Federal Register Volume 82, Issue 174 (September 11, 2017)
| Page Range | 42686-42686 | |
| FR Document | 2017-19130 |
[Federal Register Volume 82, Number 174 (Monday, September 11, 2017)] [Notices] [Page 42686] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2017-19130] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2017-N-0809] 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 (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 KYMRIAH (tisagenlecleucel), manufactured by Novartis Pharmaceuticals Corporation, meets the criteria for a priority review voucher. FOR FURTHER INFORMATION CONTACT: Gretchen Opper, 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), 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 KYMRIAH (tisagenlecleucel), manufactured by Novartis Pharmaceuticals Corporation, meets the criteria for a priority review voucher. KYMRIAH (tisagenlecleucel) is indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. 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 tohttps://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm. For further information about KYMRIAH (tisagenlecleucel), go to the Center for Biologics Evaluation and Research cellular and gene therapy products Web site at https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/default.htm. Dated: September 5, 2017. Anna K. Abram, Deputy Commissioner for Policy, Planning, Legislation, and Analysis. [FR Doc. 2017-19130 Filed 9-8-17; 8:45 am] BILLING CODE 4164-01-P
![42686 Federal Register / Vol. 82, No. 174 / Monday, September 11, 2017 / Notices
statement of the general nature of the as amended by the Food and Drug DEPARTMENT OF HEALTH AND
evidence or arguments they wish to Administration Safety and Innovation HUMAN SERVICES
present, the names and addresses of Act (FDASIA), authorizes FDA to award
proposed participants, and an priority review vouchers to sponsors of National Institutes of Health
indication of the approximate time approved rare pediatric disease product
requested to make their presentation on Government-Owned Inventions;
applications that meet certain criteria. Availability for Licensing
or before September 19, 2017. Time FDA is required to publish notice of the
allotted for each presentation may be award of the priority review voucher. AGENCY: National Institutes of Health,
limited. If the number of registrants FDA has determined that KYMRIAH HHS.
requesting to speak is greater than can (tisagenlecleucel), manufactured by ACTION: Notice.
be reasonably accommodated during the
Novartis Pharmaceuticals Corporation,
scheduled open public hearing session, SUMMARY: The invention listed below is
meets the criteria for a priority review owned by an agency of the U.S.
FDA may conduct a lottery to determine
the speakers for the scheduled open voucher. Government and is available for
public hearing session. The contact FOR FURTHER INFORMATION CONTACT: licensing to achieve expeditious
person will notify interested persons Gretchen Opper, Center for Biologics commercialization of results of
regarding their request to speak by Evaluation and Research, Food and federally-funded research and
September 20, 2017. Drug Administration, 10903 New development. Foreign patent
Persons attending FDA’s advisory Hampshire Ave., Bldg. 71, Rm. 7301, applications are filed on selected
committee meetings are advised that the Silver Spring, MD 20993–0002, 240– inventions to extend market coverage
Agency is not responsible for providing 402–7911. for companies and may also be available
access to electrical outlets. for licensing.
FDA welcomes the attendance of the SUPPLEMENTARY INFORMATION: FDA is FOR FURTHER INFORMATION CONTACT: Dr.
public at its advisory committee announcing the issuance of a priority Vince Contreras, 240–669–2823;
meetings and will make every effort to review voucher to the sponsor of an Vince.Contreras@nih.gov. Licensing
accommodate persons with disabilities. approved rare pediatric disease product information and copies of the patent
If you require accommodations due to a application. Under section 529 of the applications listed below may be
disability, please contact Serina Hunter- FD&C Act (21 U.S.C. 360ff), which was obtained by communicating with the
Thomas at least 7 days in advance of the added by FDASIA, FDA will award indicated licensing contact at the
meeting. priority review vouchers to sponsors of Technology Transfer and Intellectual
FDA is committed to the orderly approved rare pediatric disease product Property Office, National Institute of
conduct of its advisory committee applications that meet certain criteria. Allergy and Infectious Diseases, 5601
meetings. Please visit our Web site at: Fishers Lane, Rockville, MD 20852; tel.
FDA has determined that KYMRIAH
https://www.fda.gov/ 301–496–2644. A signed Confidential
(tisagenlecleucel), manufactured by
AdvisoryCommittees/ Disclosure Agreement will be required
AboutAdvisoryCommittees/ Novartis Pharmaceuticals Corporation,
meets the criteria for a priority review to receive copies of unpublished patent
ucm111462.htm for procedures on applications.
public conduct during advisory voucher. KYMRIAH (tisagenlecleucel) is
indicated for the treatment of patients SUPPLEMENTARY INFORMATION:
committee meetings. Technology description follows.
Notice of this meeting is given under up to 25 years of age with B-cell
the Federal Advisory Committee Act (5 precursor acute lymphoblastic leukemia Broadly Neutralizing Antibodies
U.S.C. app. 2). (ALL) that is refractory or in second or Against HIV–1 Directed to the CD4
Dated: September 5, 2017. later relapse. Binding Site of HIV Envelope Protein
Anna K. Abram, For further information about the Rare Description of Technology
Deputy Commissioner for Policy, Planning, Pediatric Disease Priority Review Inhibiting the ability of HIV–1, the
Legislation, and Analysis. Voucher Program and for a link to the virus that causes AIDS, to infect cells is
[FR Doc. 2017–19129 Filed 9–8–17; 8:45 am] full text of section 529 of the FD&C Act, one approach to both prevention and
BILLING CODE 4164–01–P go tohttps://www.fda.gov/ForIndustry/ treatment of HIV. Scientists at the
DevelopingProductsfor NIAID Vaccine Research Center have
RareDiseasesConditions/ isolated and characterized neutralizing
DEPARTMENT OF HEALTH AND RarePediatricDiseasePriorityVoucher antibodies (VRC01, 02, 03, and 07) that
HUMAN SERVICES Program/default.htm. For further bind to the CD4 binding site of HIV–1
information about KYMRIAH envelope glycoprotein gp120. These
Food and Drug Administration
(tisagenlecleucel), go to the Center for human monoclonal antibodies can
[Docket No. FDA–2017–N–0809] Biologics Evaluation and Research potentially be used as a therapeutic to:
cellular and gene therapy products Web (1) Treat an HIV infection, (2) decrease
Issuance of Priority Review Voucher; site at https://www.fda.gov/Biologics and prevent HIV-transmission from
Rare Pediatric Disease Product mother to infant, and (3) be effectively
BloodVaccines/CellularGeneTherapy
AGENCY: Food and Drug Administration, Products/ApprovedProducts/ combined with anti-retroviral drug
asabaliauskas on DSKBBXCHB2PROD with NOTICES
HHS. default.htm. therapy. Additionally, the antibodies
ACTION: Notice. can be used for detection of HIV–1
Dated: September 5, 2017.
infection in biological samples,
SUMMARY: The Food and Drug Anna K. Abram, including body fluids; and tissues from
Administration (FDA) is announcing the Deputy Commissioner for Policy, Planning, biopsies, autopsies, and pathology
issuance of a priority review voucher to Legislation, and Analysis. specimens.
the sponsor of a rare pediatric disease [FR Doc. 2017–19130 Filed 9–8–17; 8:45 am] VRC01 has been tested in several
product application. The Federal Food, BILLING CODE 4164–01–P phase I clinical trials for safety and
Drug, and Cosmetic Act (the FD&C Act), pharmacokinetics in infants, adults, and
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Document Created: 2017-09-09 00:04:09
Document Modified: 2017-09-09 00:04:09
| 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 | Gretchen Opper, 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. | |
| FR Citation | 82 FR 42686 |
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