2022 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee
June 29-30, 2022
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform. The Committee will meet in open session on both days to discuss the current regulatory expectations for xenotransplantation products. The discussion topics include human cells that have had ex vivo contact with animal cells, and animal organs and cells for transplantation into human subjects. On June 29, 2022, in the morning, under session 1, the Committee will meet to discuss and make recommendations on human cells that have ex vivo contact with animal cells. In the afternoon under session 2, the Committee will begin to discuss and make recommendations on animal organs and cells for transplantation into human subjects and their associated risks. On June 30, 2022, the Committee will continue to discuss and make recommendations on animal organs and cells for transplantation into human subjects.
June 9-10, 2022
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.
March 10, 2022
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform. On March 10, 2022, for Topic I, the committee will meet in open session to hear an overview of the research programs in the Gene Transfer and Immunogenicity Branch (GTIB), Division of Cellular and Gene Therapies (DCGT), Office of Tissues and Advanced Therapies (OTAT), Center for Biologics Research (CBER). After the Topic I open session, the meeting will be closed to the public for committee deliberations.