Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KEBILIDI (eladocagene exuparvovec-tneq), 95219-95220 [2024-28206]

Download as PDF 95219 Federal Register / Vol. 89, No. 231 / Monday, December 2, 2024 / Notices • Average LIHEAP household benefits, by funding type. supplemental funds, CARES Act funds, and ARPA funds. Modules 2, 2A, and 2B Required LIHEAP Performance Measures Module 3 LIHEAP Performance Measures (Optional Reporting) Modules 2, 2A, and 2B of the LPDF will continue to require the following data from each State for the Federal fiscal year: • Grant recipient information; • Energy burden targeting; • Restoration of home energy service; and • Prevention of loss of home energy. Modules 2, 2A, and 2B require reporting on households that received benefits from, respectively, non- Module 3 of the LIHEAP LPDF will continue to voluntarily collect the following additional information from each interested grant recipient for the Federal fiscal year: • Average annual energy usage; • Unduplicated number of households using supplemental heating fuel and air conditioning; • Unduplicated number of households that had restoration of home energy service, and • Unduplicated number of households that had prevention of loss of home energy. LIHEAP grant recipients will be able to compare their own results to the results for other States, as well as to regional and national results, through the Data Warehouse of the LIHEAP Performance Management website as they manage their programs. Respondents State governments, including the District of Columbia; the largest five electricity and natural gas vendors by State; the largest ten fuel oil and propane vendors by State; and State sub-grant recipients. ANNUAL BURDEN ESTIMATES LIHEAP performance data form Total number of respondents Annual number of responses per respondent 51 51 1 1 30 158.6 1,530 8,088.6 100 1 6.3 630 1,530 1 8.5 13,005 ddrumheller on DSK120RN23PROD with NOTICES1 State Grant Recipients—Module I ................................................................... State Grant Recipients—Modules II and III ..................................................... Sub-Grant Recipients (in States with sub-grant recipient managed systems)—Modules II and III ............................................................................. Energy Vendors (largest 5 electric, 5 natural gas, 10 fuel oil, and 10 propane vendors per State-average)—Modules II and III ................................ Estimated Total Annual Burden Hours: 23,253.6. DEPARTMENT OF HEALTH AND HUMAN SERVICES Comments Food and Drug Administration The Department specifically requests comments on (a) whether the proposed collection of information is necessary for the proper performance of the functions of the agency, including whether the information shall have practical utility; (b) the accuracy of the agency’s estimate of the burden of the proposed collection of information; (c) the quality, utility, and clarity of the information to be collected; and (d) ways to minimize the burden of the collection of information on respondents, including through the use of automated collection techniques or other forms of information technology. Consideration will be given to comments and suggestions submitted within 60 days of this publication. Authority: 42 U.S.C. 8629(b); 42 U.S.C. 8624(b); 42 U.S.C. 8623(c). [Docket No. FDA–2024–N–1636] Mary C. Jones, ACF/OPRE Certifying Officer. [FR Doc. 2024–28152 Filed 11–29–24; 8:45 am] BILLING CODE 4184–80–P VerDate Sep<11>2014 18:25 Nov 29, 2024 Jkt 265001 Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KEBILIDI (eladocagene exuparvovectneq) 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) 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 KEBILIDI (eladocagene exuparvovec-tneq), approved on November 13, 2024, manufactured by PTC Therapeutics Inc., meets the criteria for a priority review voucher. FOR FURTHER INFORMATION CONTACT: Myrna Hanna, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New SUMMARY: PO 00000 Frm 00052 Fmt 4703 Sfmt 4703 Average burden hours per response Annual burden hours Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993–0002, 240– 402–7911. 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 KEBILIDI (eladocagene exuparvovec-tneq), manufactured by PTC Therapeutics Inc., meets the criteria for a priority review voucher. KEBILIDI (eladocagene exuparvovectneq) is indicated for treatment of adult and pediatric patients with aromatic Lamino acid decarboxylase deficiency. 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/industry/ developing-products-rare-diseasesconditions/rare-pediatric-disease-rpddesignation-and-voucher-programs. For further information about KEBILIDI (eladocagene exuparvovec-tneq), go to the Center for Biologics Evaluation and Research’s Approved Cellular and Gene Therapy Products website at https:// www.fda.gov/vaccines-blood-biologics/ SUPPLEMENTARY INFORMATION: E:\FR\FM\02DEN1.SGM 02DEN1 95220 Federal Register / Vol. 89, No. 231 / Monday, December 2, 2024 / Notices cellular-gene-therapy-products/ approved-cellular-and-gene-therapyproducts. Dated: November 26, 2024. P. Ritu Nalubola, Associate Commissioner for Policy. [FR Doc. 2024–28206 Filed 11–29–24; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2024–N–5253] Anesthetic and Analgesic Drug Products Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments— Biologics License Application 761393 for Condoliase Injection AGENCY: Food and Drug Administration, HHS. Notice; establishment of a public docket; request for comments. ACTION: The Food and Drug Administration (FDA) announces a forthcoming public advisory committee meeting of the Anesthetic and Analgesic Drug Products Advisory Committee (the Committee). The general function of the Committee is to provide advice and recommendations to FDA on regulatory issues. The meeting will be open to the public. FDA is establishing a docket for public comment on this document. DATES: The meeting will be held on January 10, 2025, from 9 a.m. to 4:30 p.m. Eastern Time. ADDRESSES: The public may attend the meeting at the FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm. 1503), Silver Spring, MD 20993–0002. The public will also have the option to participate, and the advisory committee meeting will be heard, viewed, captioned, and recorded through an online teleconferencing and/or video conferencing platform. Answers to commonly asked questions about FDA advisory committee meetings, including information regarding special accommodations due to a disability, visitor parking, and transportation may be accessed at: https://www.fda.gov/ AdvisoryCommittees/AboutAdvisory Committees/ucm408555.htm. FDA is establishing a docket for public comment on this meeting. The docket number is FDA–2024–N–5253. The docket will close on January 9, 2025. Please note that late, untimely filed comments will not be considered. ddrumheller on DSK120RN23PROD with NOTICES1 SUMMARY: VerDate Sep<11>2014 18:25 Nov 29, 2024 Jkt 265001 The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of January 9, 2025. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are received on or before that date. Comments received on or before December 26, 2024, will be provided to the Committee. Comments received after that date will be taken into consideration by FDA. In the event that the meeting is canceled, FDA will continue to evaluate any relevant applications or information, and consider any comments submitted to the docket, as appropriate. You may submit comments as follows: Electronic Submissions Submit electronic comments in the following way: • Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https:// www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov. • If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see ‘‘Written/Paper Submissions’’ and ‘‘Instructions’’). Written/Paper Submissions Submit written/paper submissions as follows: • Mail/Hand Delivery/Courier (for written/paper submissions): Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Dockets Management Staff, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in ‘‘Instructions.’’ PO 00000 Frm 00053 Fmt 4703 Sfmt 4703 Instructions: All submissions received must include the Docket No. FDA– 2024–N–5253 for ‘‘Anesthetic and Analgesic Drug Products Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments—Biologics License Application (BLA) 761393 for Condoliase Injection.’’ Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as ‘‘Confidential Submissions,’’ publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240–402–7500. • Confidential Submissions—To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states ‘‘THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.’’ FDA will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/ blacked out, will be available for public viewing and posted on https:// www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify the information as ‘‘confidential.’’ Any information marked as ‘‘confidential’’ will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https:// www.govinfo.gov/content/pkg/FR-201509-18/pdf/2015-23389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https:// www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the ‘‘Search’’ box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500. FOR FURTHER INFORMATION CONTACT: LaToya Bonner, Center for Drug Evaluation and Research, Food and E:\FR\FM\02DEN1.SGM 02DEN1

Agencies

[Federal Register Volume 89, Number 231 (Monday, December 2, 2024)]
[Notices]
[Pages 95219-95220]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-28206]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-N-1636]


Issuance of Priority Review Voucher; Rare Pediatric Disease 
Product; KEBILIDI (eladocagene exuparvovec-tneq)

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 KEBILIDI (eladocagene exuparvovec-tneq), approved on November 13, 
2024, manufactured by PTC Therapeutics Inc., meets the criteria for a 
priority review voucher.

FOR FURTHER INFORMATION CONTACT: Myrna Hanna, 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), FDA will award priority review vouchers to sponsors of approved 
rare pediatric disease product applications that meet certain criteria. 
FDA has determined that KEBILIDI (eladocagene exuparvovec-tneq), 
manufactured by PTC Therapeutics Inc., meets the criteria for a 
priority review voucher. KEBILIDI (eladocagene exuparvovec-tneq) is 
indicated for treatment of adult and pediatric patients with aromatic 
L-amino acid decarboxylase deficiency.
    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/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs. For further information about 
KEBILIDI (eladocagene exuparvovec-tneq), go to the Center for Biologics 
Evaluation and Research's Approved Cellular and Gene Therapy Products 
website at https://www.fda.gov/vaccines-blood-biologics/

[[Page 95220]]

cellular-gene-therapy-products/approved-cellular-and-gene-therapy-
products.

    Dated: November 26, 2024.
P. Ritu Nalubola,
Associate Commissioner for Policy.
[FR Doc. 2024-28206 Filed 11-29-24; 8:45 am]
BILLING CODE 4164-01-P
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