2023 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee
October 31, 2023
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform.
On October 31, 2023, the committee will discuss and make recommendations on biologics license application (BLA) 125787 from Vertex Pharmaceuticals, Inc. for exagamglogene autotemcel (exa-cel). The applicant has requested an indication for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises.
September 27, 2023
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing 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).
May 12, 2023
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform.
On May 12, 2023, the committee will meet in open session to discuss the Biologics License Application (BLA) 125781 from 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 meeting will be open to the public.