Issuance of Priority Review Voucher; Rare Pediatric Disease Product; MIPLYFFA (Arimoclomol), 83020-83021 [2024-23646]

Download as PDF 83020 Federal Register / Vol. 89, No. 199 / Tuesday, October 15, 2024 / Notices criteria to facilitate this comparison. If a legally marketed device performs at certain levels relevant to its safety and effectiveness, and a new device meets those levels of performance for the same characteristics, FDA could find the new device as safe and effective as the legally marketed device. Instead of reviewing data from direct comparison testing between the two devices, FDA could support a finding of substantial equivalence with data demonstrating the new device meets the level of performance of an appropriate predicate device(s). Under this optional Safety and Performance Based Pathway, a submitter of an endosseous implant and endosseous implant abutment device could satisfy the requirement to compare its device with a legally marketed device by, among other things, independently demonstrating that the device’s performance meets performance criteria as established in the relevant above-listed guidance rather than using direct predicate comparison testing for some of the performance characteristics. This guidance is being implemented without prior public comment because FDA has determined that prior public participation for this guidance is not feasible or appropriate (see section 701(h)(1)(C) of the FD&C Act (21 U.S.C. II. Electronic Access Persons interested in obtaining a copy of the guidance may do so by downloading an electronic copy from the internet. A search capability for all Center for Devices and Radiological Health guidance documents is available at https://www.fda.gov/medical-devices/ device-advice-comprehensiveregulatory-assistance/guidancedocuments-medical-devices-andradiation-emitting-products. This guidance document is also available at https://www.regulations.gov or https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents. Persons unable to download an electronic copy of ‘‘Endosseous Dental Implants and Endosseous Dental Implant Abutments—Performance Criteria for Safety and Performance Based Pathway’’ may send an email request to CDRH-Guidance@fda.hhs.gov to receive an electronic copy of the document. Please use the document number GUI00021017 and complete title to identify the guidance you are requesting. III. Paperwork Reduction Act of 1995 While this guidance contains no new collection of information, it does refer to previously approved FDA collections of information. The previously approved collections of information are subject to review by the Office of Management and Budget (OMB) under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501– 3521). The collections of information in the following table have been approved by OMB: OMB Control No. 21 CFR part; guidance; or FDA form Topic 807, subpart E ............................................................................ ‘‘Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program’’. Premarket notification ................................................................. Q-submissions and Early Payor Feedback Request Programs for Medical Devices. Dated: October 4, 2024. Kimberlee Trzeciak, Deputy Commissioner for Policy, Legislation, and International Affairs. [FR Doc. 2024–23716 Filed 10–11–24; 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; MIPLYFFA (Arimoclomol) khammond on DSKJM1Z7X2PROD with NOTICES 371(h)(1)(C)) and 21 CFR 10.115(g)(2)). FDA has determined that this guidance document presents a less burdensome policy that is consistent with public health. Although this policy is being implemented immediately without prior comment, it remains subject to comment in accordance with FDA’s good guidance practices regulation (21 CFR 10.115(g)(3)(D)). FDA will consider all comments received and revise the guidance document as appropriate. This guidance is being issued consistent with FDA’s good guidance practices regulation (21 CFR 10.115). The guidance represents the current thinking of FDA on Endosseous Dental Implants and Endosseous Dental Implant Abutments—Performance Criteria for Safety and Performance Based Pathway. 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. AGENCY: Food and Drug Administration, HHS. ACTION: The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease SUMMARY: 16:35 Oct 11, 2024 FOR FURTHER INFORMATION CONTACT: Cathryn Lee, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–1394. 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), FDA will SUPPLEMENTARY INFORMATION: Notice. VerDate Sep<11>2014 product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) 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 MIPLYFFA (arimoclomol), approved on September 20, 2024, manufactured by Zevra Denmark AS, meets the criteria for a priority review voucher. Jkt 265001 PO 00000 Frm 00052 Fmt 4703 Sfmt 4703 0910–0120 0910–0756 award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined that MIPLYFFA (arimoclomol), manufactured by Zevra Denmark AS, meets the criteria for a priority review voucher. MIPLYFFA (arimoclomol) is indicated for the treatment of adults and pediatric patients 2 years of age and older with Niemann-Pick disease type C. 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 MIPLYFFA (arimoclomol), go to the ‘‘Drugs@FDA’’ website at https:// www.accessdata.fda.gov/scripts/cder/ daf/. E:\FR\FM\15OCN1.SGM 15OCN1 Federal Register / Vol. 89, No. 199 / Tuesday, October 15, 2024 / Notices Dated: October 8, 2024. Eric Flamm, Acting Associate Commissioner for Policy. for electronic access to the Authorization. [FR Doc. 2024–23646 Filed 10–11–24; 8:45 am] Food and Drug Administration Tami Belouin, 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: [Docket No. FDA–2021–N–0335] I. Background FOR FURTHER INFORMATION CONTACT: BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Revocation of Emergency Use of a Biological Product During the COVID– 19 Pandemic; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA or Agency) is announcing the revocation of the Emergency Use Authorization (EUA) (the Authorization) issued to Janssen Biotech, Inc. (Janssen), for the Janssen COVID–19 Vaccine. FDA revoked the Authorization on June 1, 2023, under the Federal Food, Drug, and Cosmetic Act (FD&C Act). The revocation, which includes an explanation of the reasons for the revocation, is reprinted in this document. SUMMARY: The Authorization is revoked as of June 1, 2023. ADDRESSES: Submit written requests for a single copy of the revocation to the Office of Communication, Outreach and Development, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993–0002. Send one self-addressed adhesive label to assist that office in processing your request or include a Fax number to which the revocation may be sent. See the SUPPLEMENTARY INFORMATION section khammond on DSKJM1Z7X2PROD with NOTICES DATES: VerDate Sep<11>2014 16:35 Oct 11, 2024 Jkt 265001 Section 564 of the FD&C Act (21 U.S.C. 360bbb–3) allows FDA to strengthen the public health protections against biological, chemical, nuclear, and radiological agents. Among other things, section 564 of the FD&C Act allows FDA to authorize the use of an unapproved medical product or an unapproved use of an approved medical product in certain situations. On February 27, 2021, FDA issued an Authorization (EUA 27205) to Janssen for the Janssen COVID–19 Vaccine, subject to the terms of the Authorization. Notice of the issuance of the Authorization was published in the Federal Register on May 27, 2021 (86 FR 28608), as required by section 564(h)(1) of the FD&C Act. Subsequent updates to the Authorization were made available on FDA’s website. The authorization of a biological product for emergency use under section 564 of the FD&C Act may, pursuant to section 564(g)(2) of the FD&C Act, be revoked when the criteria under section 564(c) of the FD&C Act for issuance of such authorization are no longer met (section 564(g)(2)(B) of the FD&C Act), or other circumstances make such revocation appropriate to protect the public health or safety (section 564(g)(2)(C) of the FD&C Act). II. EUA Revocation Request In a request received by FDA on May 22, 2023, Janssen requested withdrawal PO 00000 Frm 00053 Fmt 4703 Sfmt 4703 83021 of, and on June 1, 2023, FDA revoked, the Authorization for the Janssen COVID–19 Vaccine. Janssen notified FDA that the last lots of the Janssen COVID–19 Vaccine purchased by the U.S. Government have expired, that there is no demand for new lots of the Janssen COVID–19 Vaccine in the United States, and that it does not intend to update the strain composition of this vaccine to address emerging variants. Based on FDA’s understanding that Janssen does not intend to offer the Janssen COVID–19 Vaccine in the United States under the EUA anymore and Janssen’s request that FDA revoke the EUA for the Janssen COVID–19 Vaccine, FDA has determined that it is appropriate to revoke this Authorization to protect the public health or safety. III. The Revocation Having concluded that the criteria for revocation of the Authorization under section 564(g)(2)(C) of the FD&C Act are met, FDA has revoked the EUA for the Janssen COVID–19 Vaccine. Although FDA revoked the Authorization for the Janssen COVID–19 Vaccine on June 1, 2023, as was publicly announced on the Agency’s website, publication of notice of this revocation in the Federal Register was inadvertently delayed. The revocation in its entirety follows and provides an explanation of the reasons for revocation, as required by section 564(h)(1) of the FD&C Act. IV. Electronic Access An electronic version of this document and the full text of the Authorizations and revocation are available on the internet at https:// www.fda.gov/emergency-preparednessand-response/mcm-legal-regulatoryand-policy-framework/emergency-useauthorization. BILLING CODE 4164–01–P E:\FR\FM\15OCN1.SGM 15OCN1

Agencies

[Federal Register Volume 89, Number 199 (Tuesday, October 15, 2024)]
[Notices]
[Pages 83020-83021]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-23646]


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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; MIPLYFFA (Arimoclomol)

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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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) 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 MIPLYFFA (arimoclomol), approved on September 20, 2024, 
manufactured by Zevra Denmark AS, meets the criteria for a priority 
review voucher.

FOR FURTHER INFORMATION CONTACT: Cathryn Lee, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-1394.

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), FDA will award priority review vouchers to sponsors of approved 
rare pediatric disease product applications that meet certain criteria. 
FDA has determined that MIPLYFFA (arimoclomol), manufactured by Zevra 
Denmark AS, meets the criteria for a priority review voucher. MIPLYFFA 
(arimoclomol) is indicated for the treatment of adults and pediatric 
patients 2 years of age and older with Niemann-Pick disease type C.
    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 MIPLYFFA (arimoclomol), go to the ``Drugs@FDA'' 
website at https://www.accessdata.fda.gov/scripts/cder/daf/.


[[Page 83021]]


    Dated: October 8, 2024.
Eric Flamm,
Acting Associate Commissioner for Policy.
[FR Doc. 2024-23646 Filed 10-11-24; 8:45 am]
BILLING CODE 4164-01-P

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