Issuance of Priority Review Voucher; Rare Pediatric Disease Product, 71511 [2016-24947]

Download as PDF jstallworth on DSK7TPTVN1PROD with NOTICES Federal Register / Vol. 81, No. 200 / Monday, October 17, 2016 / Notices security check procedures will be performed. For parking and security information, please refer to https:// www.fda.gov/AboutFDA/ WorkingatFDA/BuildingsandFacilities/ WhiteOakCampusInformation/ ucm241740.htm. FOR FURTHER INFORMATION CONTACT: Audrey Thomas, Office of Regulatory Science and Innovation, Office of the Chief Scientist, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 1, Rm. 4220, Silver Spring, MD 20993–0002, 301–796–3520, Audrey.Thomas@fda.hhs.gov. SUPPLEMENTARY INFORMATION: The purpose of this public workshop is to provide an opportunity for stakeholders, including scientists from government, academia, and industry, patient advocacy groups, clinicians, pharmacists, and the general public to discuss marketed generic drugs. Generic drugs account for 88 percent of prescriptions in the United States. In light of the significant contributions of generic drugs to public health, it is important that tools are developed to monitor marketed generic drugs to ensure that they have the same safety and effectiveness as their reference listed drug. Specifically, this workshop will include presentations on: (1) Current generic drug surveillance practices at FDA and in industry, (2) public perception of generic drug quality and effectiveness, (3) generic drug substitution studies in patients, and (4) development of methods and tools to conduct postmarket surveillance of generic drugs. The workshop will include four panel sessions for interaction and discussion among the speakers and attendees. Agenda: The agenda is available at https://www.fda.gov/scienceresearch/ specialtopics/regulatoryscience/ ucm521545.htm. Registration: There is no registration fee to attend this public workshop. Seats are limited and registration will be on a first-come, first-served basis. Advance registration is required and is online only at https://www.fda.gov/ scienceresearch/specialtopics/ regulatoryscience/ucm521545.htm. There will be no day-of, onsite registration. Streaming Webcast of the Public Workshop: This public workshop will also be Webcast. There is no registration fee for access to the workshop via the Webcast, but registration is still required. Information regarding registration and access to the Webcast link is available at https://www.fda.gov/ scienceresearch/specialtopics/ regulatoryscience/ucm521545.htm. If VerDate Sep<11>2014 14:22 Oct 14, 2016 Jkt 241001 you have never attended a Connect Pro event before, test your connection at https://collaboration.fda.gov/common/ help/en/support/meeting_test.htm. To get a quick overview of the Connect Pro program, visit https://www.adobe.com/ go/connectpro_overview. (FDA has verified these Web site addresses, but FDA is not responsible for any subsequent changes to the Web sites after this document publishes in the Federal Register.) Accommodations: Attendees are responsible for their own hotel accommodations. If you need special accommodations while at FDA’s White Oak Campus due to a disability, please contact Shari Solomon at Shari.Solomon@fda.hhs.gov at least 7 days in advance. Dated: October 11, 2016. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2016–25004 Filed 10–14–16; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2014–N–0229] Food and Drug Administration, HHS. ACTION: Dated: October 6, 2016. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2016–24947 Filed 10–14–16; 8:45 am] DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration Notice. [Docket No. FDA–2014–D–0901] 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 rare pediatric disease product applications that meet certain criteria. FDA has determined that EXONDYS 51 (eteplirsen), manufactured by Sarepta Therapeutics, meets the criteria for a priority review voucher. FOR FURTHER INFORMATION CONTACT: Larry Bauer, Rare Diseases Program, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–4842, FAX: 301–796–9858, email: larry.bauer@fda.hhs.gov. SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. SUMMARY: PO 00000 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 rare pediatric disease product applications that meet certain criteria. FDA has determined that EXONDYS 51 (eteplirsen), manufactured by Sarepta Therapeutics, meets the criteria for a priority review voucher. EXONDYS 51 (eteplirsen) is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 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/ DevelopingProductsforRareDiseases Conditions/RarePediatricDiseasePriority VoucherProgram/default.htm. For further information about EXONDYS 51 (eteplirsen) go to the ‘‘Drugs@FDA’’ Web site at https://www.accessdata.fda.gov/ scripts/cder/drugsatfda/index.cfm. BILLING CODE 4164–01–P Issuance of Priority Review Voucher; Rare Pediatric Disease Product AGENCY: 71511 Frm 00037 Fmt 4703 Sfmt 4703 Abbreviated New Drug Application Submissions—Prior Approval Supplements Under Generic Drug User Fee Amendments; Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a guidance for industry entitled ‘‘ANDA Submissions—Prior Approval Supplements Under GDUFA.’’ The Generic Drug User Fee Amendments of 2012 (GDUFA) enables FDA to assess user fees to fund critical and measurable improvements to FDA’s generic drugs program. This guidance is intended to assist applicants preparing to submit to FDA prior approval supplements (PASs) and amendments to PASs for abbreviated new drug applications (ANDAs). It describes FDA’s performance metric goals for PASs and clarifies how FDA will handle a PAS SUMMARY: E:\FR\FM\17OCN1.SGM 17OCN1

Agencies

[Federal Register Volume 81, Number 200 (Monday, October 17, 2016)]
[Notices]
[Page 71511]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-24947]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0229]

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 rare pediatric disease
product applications that meet certain criteria. FDA has determined
that EXONDYS 51 (eteplirsen), manufactured by Sarepta Therapeutics,
meets the criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT: Larry Bauer, Rare Diseases Program,
Center for Drug Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4842,
FAX: 301-796-9858, email: larry.bauer@fda.hhs.gov.

SUPPLEMENTARY INFORMATION: FDA is announcing the issuance of a priority
review voucher to the sponsor of a 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 rare pediatric disease product applications that meet
certain criteria. FDA has determined that EXONDYS 51 (eteplirsen),
manufactured by Sarepta Therapeutics, meets the criteria for a priority
review voucher. EXONDYS 51 (eteplirsen) is indicated for the treatment
of Duchenne muscular dystrophy (DMD) in patients who have a confirmed
mutation of the DMD gene that is amenable to exon 51 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 EXONDYS 51 (eteplirsen) go to the ``Drugs@FDA'' Web
site at https://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm.

Dated: October 6, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-24947 Filed 10-14-16; 8:45 am]
BILLING CODE 4164-01-P

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