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Advisory Committee Meeting | Virtual

Event Title
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Announcement
September 27, 2023

Scheduled

Date:
September 27, 2023
Time:
10:00 AM - 6:00 PM ET

What is an advisory committee?

Advisory committees provide independent expert advice to the FDA on broad scientific topics or on certain products to help the agency make sound decisions based on the available science. Advisory committees make non-binding recommendations to the FDA, which generally follows the recommendations but is not legally bound to do so. Please see, "Advisory Committees Give FDA Critical Advice and the Public a Voice," for more information.


Please note that due to the impact of this COVID-19 pandemic, all meeting participants will be joining this advisory committee meeting via an online teleconferencing platform.

The online web conference meeting will be available at the following: 



Agenda
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing and/or video conferencing platform. On September 27, 2023, the committee will meet in open session to discuss and make recommendations on biologics license application (BLA) 125782 from BrainStorm Therapeutics, Inc. for debamestrocel (autologous bone marrow-derived mesenchymal stromal cells induced to secrete neurotrophic factors). The applicant has requested an indication for the treatment of amyotrophic lateral sclerosis (ALS).


Meeting Materials
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 at the time of the advisory committee meeting, and the background material will be posted on FDA’s website after the meeting. Background material is available at the Advisory Committee Calendar. Scroll down to the appropriate advisory committee meeting link. The meeting will include slide presentations with audio components to allow the presentation of materials in a manner that most closely resembles an in-person advisory committee meeting.


Public Participation Information
Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee.

Oral presentations from the public will be scheduled between approximately 1:40 p.m. and 2:40 p.m. Eastern Time on September 27, 2023.

FDA is establishing a docket for public comment on this meeting.

The docket number is FDA-2023-N-2608.

The docket will close on September 26, 2023. Submit either electronic or written comments on this public meeting on or before September 26, 2023. Please note that late, untimely filed comments will not be considered. Electronic comments must be submitted on or before September 26, 2023. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of September 26, 2023. 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 September 20, 2023, 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 as follows:

  • 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-2023-N-2608 for “Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments.” 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 Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION, CONTACT: Marie DeGregorio or Cicely Reese, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1246, Silver Spring, MD 20993-0002, CBERCTGTAC@fda.hhs.gov, 240 701-9119, or FDA Advisory Committee Information Line, 1-800-741-8138 (301-443-0572 in the Washington, DC area). 


Oral Presentations
Oral presentations from the public will be scheduled between approximately 1:40 p.m. and 2:40 p.m. Eastern Time on September 27, 2023.

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 12 p.m. Eastern Time on September 12, 2023.

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 6 pm Eastern Time on September 14, 2023.


Webcast Information
CBER plans to provide a free of charge live webcast of the Cellular, Tissue, and Gene Therapies Advisory Committee meeting: 

The online web conference meeting will be available at the following link:

If there are instances where the webcast transmission is not successful, staff will work to re-establish the transmission as soon as possible. 


Contact Information

  • Marie DeGregorio or Cicely Reese CBERCTGTAC@fda.hhs.gov
  • FDA Advisory Committee Information Line: 
    1-800-741-8138 (301-443-0572 in the Washington, DC area)
    Please call the Information Line for up-to-date information on this meeting.
  • For press inquiries, please contact the Office of Media Affairs at fdaoma@fda.hhs.gov or 301-796-4540.

Official FR Notice


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 the agency’s website or call the committee’s Designated Federal Officer (see Contact Information) to learn about possible modifications before coming to the meeting.

Persons attending FDA’s advisory committee meetings are advised that the agency is not responsible for providing access to electrical outlets. 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 the committee’s Designated Federal Officer (see Contact Information) at least 7 days in advance of the meeting.

Answers to commonly asked questions including information regarding special accommodations due to a disability may be accessed at: Common Questions and Answers about FDA Advisory Committee Meetings.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our Web site at Public Conduct During FDA Advisory Committee Meetings 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. app.2).


Event Materials

Title File Type/Size Source Organization
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Dr. Jun Li- Acknowledgement of Financial Interests pdf (138.98 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Dr. Jun Li- Waiver to Allow Participation pdf (254.10 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Draft Roster pdf (298.68 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Briefing Document- Sponsor pdf (3.47 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Draft Agenda pdf (338.94 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Briefing Document- FDA pdf (2.36 MB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Voting Questions pdf (126.53 KB) FDA
Cellular, Tissue and Gene Therapies Advisory Committee September 27, 2023 Meeting Briefing Document- FDA- Errata pdf (182.66 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Presentation- Sponsor- NurOwn for Treatment of ALS pdf (1.85 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Presentation- BLA 125782 Application for debamestrocel (MSC-NTF) for Treatment of ALS pdf (2.23 MB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Presentation- FDA Overview of BLA 125782 pdf (1.08 MB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Presentation- Some Guiding Principles When Contemplating Stem Cell-Based Approaches to Neurological Disease pdf (26.15 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Transcript pdf (1.51 MB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Final Roster- Revised pdf (178.44 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Final Agenda pdf (379.37 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 27, 2023 Meeting Summary Minutes pdf (244.94 KB) FDA

 
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