FDA Advisory Committees - FDA Center for Biologics Evaluation and Research (CBER) - Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC)
Upcoming Meetings
Past Meetings
The USFDA has scheduled a Cellular, Tissue, and Gene Therapies Advisory Committee
[CTGTAC] on October 31, 2023, to discuss and make recommendations on biologics license application (BLA) 125787 from Vertex Pharmaceutical’s Inc. Exagamglogene autotemcel (exa-cel) for the treatment of Sickle cell disease (SCD) in patients 12 years and older with Recurrent Vaso-occlusive crises (VOCs).
The USFDA has scheduled a Cellular, Tissue, and Gene Therapies Advisory Committee
[CTGTAC] on September 27, 2023, to discuss and make recommendations on biologics license application (BLA) 125782 from BrainStorm Therapeutics, Inc. for debamestrocel NurOwn® (autologous bone marrow-derived mesenchymal stromal cells induced to secrete neurotrophic factors (MSC-NTF). BrainStorm Therapeutics has requested an indication for the treatment of amyotrophic lateral sclerosis (ALS).
The Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) has scheduled a meeting on Friday May 12, 2023. During the meeting, the committee will review Biologic license application (BLA 125781) submitted by Sarepta Therapeutics, Inc. for delandistrogene moxeparvovec with the requested indication for the treatment of ambulatory patients with Duchenne Muscular Dystrophy (DMD) with a confirmed mutation in the DMD gene.
The Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) on June 10th, 2022 (day 2) will review a Biologic license application (BLA) submitted by Bluebird Bio; BLA 125717 for betibeglogene autotemcel for the treatment of patients with β-thalassemia who require regular red blood cell transfusions cross all genotypes who require regular red blood cell (RBC).
The Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) on Thursday, June 9th (day 1) will review a Biologic license application (BLA) submitted by Bluebird Bio namely, BLA 125755 for elivaldogene autotemcel for the treatment of patients less than 18 years of age with early cerebral adrenoleukodystrophy.
The Committee unanimously supported, by a vote of 16-Yes to 0-No, with no abstentions, the safety and efficacy of voretigene neparvovec by Spark Therapeutics, Inc. (Spark). The proposed indication is the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy. The proposed trade name is Luxturna. The FDA’s goal date for a decision (PDUFA date) is January 12, 2018.
The Committee heard updates of CBER research programs.
Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC)
CTGTAC reviews and evaluates available data relating to the safety, effectiveness, and appropriate use of
- human cells
- human tissues
- gene transfer therapies, and
- xenotransplantation products
which are intended for
- transplantation
- implantation
- infusion, and
- transfer
in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair or replacement of tissues for various conditions.
On Friday, May 8, 2020 the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) will meet by teleconference to hear an overview and updates of research programs in the Tumor Vaccines and Biotechnology Branch (TVBB) and Cellular and Tissue Therapy Branch (CTTB), Division of Cellular and Gene Therapies (DCGT), Office of Tissues and Advanced Therapies (OTAT), CBER, FDA.