Day 2 June 10:

Agenda
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform. The committee will discuss two biologics license applications (BLAs) from bluebird bio, Inc.: 1) BLA 125755 for elivaldogene autotemcel (autologous CD34+ stem cells genetically modified with a lentiviral vector to contain an adenosine triphosphate binding cassette, sub-family D, member 1(ABCD1) gene which encodes a functional adrenoleukodystrophy protein (ALDP)); the applicant has requested an indication for the “treatment of patients less than 18 years of age with early cerebral adrenoleukodystrophy who do not have an available and willing HLA-matched sibling hematopoietic stem cell (HSC) donor” 2) BLA 125717 for betibeglogene autotemcel (autologous CD34+ stem cells genetically modified with a lentiviral vector to contain a gene encoding functional beta-globin); the applicant has requested an indication for the “treatment of patients with β-thalassemia who require regular red blood cell transfusions.”

The morning session of June 9, 2022, will include presentations of the effectiveness and product specific safety results from the clinical trials in BLA 125755. The afternoon session will include presentations of safety concerns relevant to both products, followed by committee discussion of BLA 125755. On June 10, 2022, the morning session will include presentations of the effectiveness and product-specific safety results from the clinical trials in BLA 125717. The afternoon session will include committee discussion of BLA 125717.

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, any background material will be made publicly available at the time of the advisory committee meeting, and additional materials will be posted on FDA's website after the meeting.

Materials for this meeting will be available at the Cellular, Tissue, and Gene Therapies Advisory Committee meeting main page.

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.

FDA is establishing a docket for public comment on this meeting. The docket number is FDA-2022-N-0470.

The docket will close on June 8, 2022. Submit either electronic or written comments on this public meeting on or before June 8, 2022. Please note that late, untimely filed comments will not be considered. Electronic comments must be submitted on or before June 8, 2022. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of June 8, 2022. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are postmarked or the delivery service acceptance receipt is on or before that date.

Comments received on or before June 2, 2022, 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-2022-N-0470. 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: Christina Vert or Tonica Burke, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1244, Silver Spring, MD 20993-0002, ctgtac@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 the FDA’s website at 2022 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee and scroll down to the appropriate advisory committee meeting link, or call the advisory committee information line to learn about possible modifications before coming to the meeting.

Oral presentations
Oral presentations from the public will be scheduled between approximately 1 p.m. and 2 p.m. Eastern Time on June 9, 2022, and between approximately 1 p.m. and 2 p.m. Eastern Time on June 10, 2022.

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 May 25, 2022.

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 May 26, 2022.

Webcast Information
CBER plans to provide a free of charge, live webcast of the Cellular, Tissue and Gene Therapies Advisory Committee meeting. If there are instances where the webcast transmission is not successful; staff will work to re-establish the transmission as soon as possible.

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

• Day 1 June 9 link: https://youtu.be/RvtTK3KNl5g
• Day 2 June 10 link: https://youtu.be/Eo2BXnGienc

Contact Information

• Christina Vert, by e-mail: ctgtac@fda.hhs.gov
• Tonica Burke, by e-mail: ctgtac@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.

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 meeting main page or call the appropriate advisory committee hot line/phone line to learn about possible modifications before coming to the meeting.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our website 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).

Official FR Notice