Genetic Metabolic Diseases Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments-New Drug Application 214927, for Arimoclomol, 56756-56757 [2024-15127]

Download as PDF 56756 Federal Register / Vol. 89, No. 132 / Wednesday, July 10, 2024 / Notices Number: CMS–R–284 (OMB control number: 0938–0345); Frequency: Quarterly and monthly; Affected Public: State, Local, or Tribal Governments; Number of Respondents: 54; Total Annual Responses: 648; Total Annual Hours: 7,290. (For policy questions regarding this collection contact Connie Gibson at 410–786–0755.) William N. Parham, III, Director, Division of Information Collections and Regulatory Impacts, Office of Strategic Operations and Regulatory Affairs. [FR Doc. 2024–15152 Filed 7–9–24; 8:45 am] BILLING CODE 4120–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2024–N–2930] Genetic Metabolic Diseases Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments—New Drug Application 214927, for Arimoclomol 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 Genetic Metabolic Diseases 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 August 2, 2024, from 9 a.m. to 6 p.m. Eastern Time. ADDRESSES: FDA and invited participants 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 have the option to participate via an online teleconferencing and/or video conferencing platform, 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 ddrumheller on DSK120RN23PROD with NOTICES1 SUMMARY: VerDate Sep<11>2014 18:33 Jul 09, 2024 Jkt 262001 be accessed at: https://www.fda.gov/ AdvisoryCommittees/ AboutAdvisoryCommittees/ ucm408555.htm. FDA is establishing a docket for public comment on this meeting. The docket number is FDA–2024–N–2930. The docket will close on August 1, 2024. Please note that late, untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of August 1, 2024. 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 July 25, 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 cancelled, 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 PO 00000 Frm 00042 Fmt 4703 Sfmt 4703 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.’’ Instructions: All submissions received must include the Docket No. FDA– 2024–N–2930 for ‘‘Genetic Metabolic Diseases Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments— Arimoclomol.’’ 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 E:\FR\FM\10JYN1.SGM 10JYN1 ddrumheller on DSK120RN23PROD with NOTICES1 Federal Register / Vol. 89, No. 132 / Wednesday, July 10, 2024 / Notices Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500. FOR FURTHER INFORMATION CONTACT: Moon Choi, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 31, Rm. 2417, Silver Spring, MD 20993–0002, 301–796–2894, email: GeMDAC@fda.hhs.gov, or FDA Advisory Committee Information Line, 1–800–741–8138 (301–443–0572 in the Washington, DC area). A notice in the Federal Register about last-minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check FDA’s website at https://www.fda.gov/ AdvisoryCommittees/default.htm and scroll down to the appropriate advisory committee meeting link, or call the advisory committee information line to learn about possible modifications before the meeting. SUPPLEMENTARY INFORMATION: Agenda: The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing and/or video conferencing platform. The Committee will discuss new drug application 214927, for arimoclomol, submitted by Zevra Denmark A/S, for the treatment of adults and pediatric patients 2 years of age and older with Niemann-Pick disease type C. FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its website prior to the meeting, the background material will be made publicly available on FDA’s website at the time of the advisory committee meeting. Background material and the link to the online teleconference and/or video conference meeting will be available at https:// www.fda.gov/AdvisoryCommittees/ Calendar/default.htm. Scroll down to the appropriate advisory committee meeting link. For online participants the meeting will include slide presentations with audio and video components to allow the presentation of materials in a manner that most closely resembles an in-person advisory committee meeting. Procedure: Interested persons may present data, information, or views, orally or in writing, on issues pending before the Committee. All electronic and written submissions to the Docket (see ADDRESSES) on or before July 25, 2024, will be provided to the Committee. Oral presentations from the public will be scheduled between approximately between 2:25 p.m. and 3:25 p.m. Eastern VerDate Sep<11>2014 18:33 Jul 09, 2024 Jkt 262001 Time. Those individuals interested in making formal oral presentations should notify the contact person and submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation on or before July 17, 2024. Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session. The contact person will notify interested persons regarding their request to speak by July 18, 2024. For press inquiries, please contact the Office of Media Affairs at fdaoma@ fda.hhs.gov or 301–796–4540. FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with disabilities. If you require accommodations due to a disability, please contact Moon Choi (see FOR FURTHER INFORMATION CONTACT) at least 7 days in advance of the meeting. FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our website at: https://www.fda.gov/ AdvisoryCommittees/ AboutAdvisoryCommittees/ ucm111462.htm for procedures on public conduct during advisory committee meetings. Notice of this meeting is given under the Federal Advisory Committee Act (5 U.S.C. 1001 et seq.). This meeting notice also serves as notice that, pursuant to 21 CFR 10.19, the requirements in 21 CFR 14.22(b), (f), and (g) relating to the location of advisory committee meetings are hereby waived to allow for this meeting to take place both in-person and using an online meeting platform. This waiver is in the interest of allowing greater transparency and opportunities for public participation, in addition to convenience for advisory committee members, speakers, and guest speakers. The conditions for issuance of a waiver under 21 CFR 10.19 are met. Dated: July 5, 2024. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2024–15127 Filed 7–9–24; 8:45 am] BILLING CODE 4164–01–P PO 00000 Frm 00043 Fmt 4703 Sfmt 4703 56757 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2024–N–0001] Workshop To Consider Artificial Intelligence in Drug and Biological Product Development; Public Workshop AGENCY: Food and Drug Administration, HHS. ACTION: Notice of public workshop. The Food and Drug Administration (FDA, the Agency, or we) is announcing the following public workshop titled ‘‘Artificial Intelligence (AI) in Drug & Biological Product Development.’’ Convened and supported by a cooperative agreement between FDA and the Clinical Trials Transformation Initiative (CTTI), the purpose of the public workshop is to bring drug developers and AI experts together to discuss guiding principles for the responsible use of AI in the development of safe and effective drugs and biological products. The workshop format will include presentations and panel discussions. SUMMARY: The public workshop will be held virtually and in-person on August 6, 2024, from 10 a.m. to 5:30 p.m. Eastern Time. See the SUPPLEMENTARY INFORMATION section for registration date and information. DATES: Participants can join the public workshop either virtually using the Zoom platform or in person at the FDA Great Room, located at 10903 New Hampshire Ave., Silver Spring, MD, 20993. The link for the public workshop will be sent to registrants upon registration. ADDRESSES: FOR FURTHER INFORMATION CONTACT: Marsha Samson, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 6330, Silver Spring, MD 20993–0002, 301– 837–7407, Marsha.Samson@ fda.hhs.gov. SUPPLEMENTARY INFORMATION: I. Background This public workshop will convene experts in AI including drug sponsors, academia, and technology organizations to discuss guiding principles that are being applied by innovators to promote the responsible use of AI in the development of safe and effective drugs. E:\FR\FM\10JYN1.SGM 10JYN1

Agencies

[Federal Register Volume 89, Number 132 (Wednesday, July 10, 2024)]
[Notices]
[Pages 56756-56757]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-15127]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-N-2930]


Genetic Metabolic Diseases Advisory Committee; Notice of Meeting; 
Establishment of a Public Docket; Request for Comments--New Drug 
Application 214927, for Arimoclomol

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; establishment of a public docket; request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) announces a forthcoming 
public advisory committee meeting of the Genetic Metabolic Diseases 
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 August 2, 2024, from 9 a.m. to 6 
p.m. Eastern Time.

ADDRESSES: FDA and invited participants 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 have the option to participate via an online 
teleconferencing and/or video conferencing platform, 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/AboutAdvisoryCommittees/ucm408555.htm.
    FDA is establishing a docket for public comment on this meeting. 
The docket number is FDA-2024-N-2930. The docket will close on August 
1, 2024. Please note that late, untimely filed comments will not be 
considered. The https://www.regulations.gov electronic filing system 
will accept comments until 11:59 p.m. Eastern Time at the end of August 
1, 2024. 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 July 25, 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 cancelled, 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.''
    Instructions: All submissions received must include the Docket No. 
FDA-2024-N-2930 for ``Genetic Metabolic Diseases Advisory Committee; 
Notice of Meeting; Establishment of a Public Docket; Request for 
Comments--Arimoclomol.'' 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-2015-09-18/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

[[Page 56757]]

Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION CONTACT: Moon Choi, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 31, Rm. 2417, Silver Spring, MD 20993-0002, 301-796-2894, email: 
[email protected], or FDA Advisory Committee Information Line, 1-800-
741-8138 (301-443-0572 in the Washington, DC area). A notice in the 
Federal Register about last-minute modifications that impact a 
previously announced advisory committee meeting cannot always be 
published quickly enough to provide timely notice. Therefore, you 
should always check FDA's website at https://www.fda.gov/AdvisoryCommittees/default.htm and scroll down to the appropriate 
advisory committee meeting link, or call the advisory committee 
information line to learn about possible modifications before the 
meeting.

SUPPLEMENTARY INFORMATION: 
    Agenda: The meeting presentations will be heard, viewed, captioned, 
and recorded through an online teleconferencing and/or video 
conferencing platform.
    The Committee will discuss new drug application 214927, for 
arimoclomol, submitted by Zevra Denmark A/S, for the treatment of 
adults and pediatric patients 2 years of age and older with Niemann-
Pick disease type C.
    FDA intends to make background material available to the public no 
later than 2 business days before the meeting. If FDA is unable to post 
the background material on its website prior to the meeting, the 
background material will be made publicly available on FDA's website at 
the time of the advisory committee meeting. Background material and the 
link to the online teleconference and/or video conference meeting will 
be available at https://www.fda.gov/AdvisoryCommittees/Calendar/default.htm. Scroll down to the appropriate advisory committee meeting 
link. For online participants the meeting will include slide 
presentations with audio and video components to allow the presentation 
of materials in a manner that most closely resembles an in-person 
advisory committee meeting.
    Procedure: Interested persons may present data, information, or 
views, orally or in writing, on issues pending before the Committee. 
All electronic and written submissions to the Docket (see ADDRESSES) on 
or before July 25, 2024, will be provided to the Committee. Oral 
presentations from the public will be scheduled between approximately 
between 2:25 p.m. and 3:25 p.m. Eastern Time. Those individuals 
interested in making formal oral presentations should notify the 
contact person and submit a brief statement of the general nature of 
the evidence or arguments they wish to present, the names and addresses 
of proposed participants, and an indication of the approximate time 
requested to make their presentation on or before July 17, 2024. Time 
allotted for each presentation may be limited. If the number of 
registrants requesting to speak is greater than can be reasonably 
accommodated during the scheduled open public hearing session, FDA may 
conduct a lottery to determine the speakers for the scheduled open 
public hearing session. The contact person will notify interested 
persons regarding their request to speak by July 18, 2024.
    For press inquiries, please contact the Office of Media Affairs at 
[email protected] or 301-796-4540.
    FDA welcomes the attendance of the public at its advisory committee 
meetings and will make every effort to accommodate persons with 
disabilities. If you require accommodations due to a disability, please 
contact Moon Choi (see FOR FURTHER INFORMATION CONTACT) at least 7 days 
in advance of the meeting.
    FDA is committed to the orderly conduct of its advisory committee 
meetings. Please visit our website at: https://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm111462.htm for procedures 
on public conduct during advisory committee meetings.
    Notice of this meeting is given under the Federal Advisory 
Committee Act (5 U.S.C. 1001 et seq.). This meeting notice also serves 
as notice that, pursuant to 21 CFR 10.19, the requirements in 21 CFR 
14.22(b), (f), and (g) relating to the location of advisory committee 
meetings are hereby waived to allow for this meeting to take place both 
in-person and using an online meeting platform. This waiver is in the 
interest of allowing greater transparency and opportunities for public 
participation, in addition to convenience for advisory committee 
members, speakers, and guest speakers. The conditions for issuance of a 
waiver under 21 CFR 10.19 are met.

    Dated: July 5, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-15127 Filed 7-9-24; 8:45 am]
BILLING CODE 4164-01-P

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