FDA Advisory Committees - FDA Center for Biologics Evaluation and Research (CBER) - Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC)
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 research programs at FDA’s Center for Biologics Evaluation and Research (CBER).
The Committee heard updates of CDER research programs.
The Committee heard updates of research programs in the Tumor Vaccines and Biotechnology Branch and the Cellular and Tissue Therapy Branch of the Division of Cellular and Gene Therapies, Office of Cellular, Tissue and Gene Therapy, Center for Biologics Evaluation and Research, FDA
The Committees jointly supported, by a vote of 22-Yes to 1-No, the safety and efficacy of talimogene laherparepvec (T-Vec), submitted by Amgen, Inc. (Amgen). T-Vec is an oncolytic immunotherapy proposed for the treatment of patients with injectable regionally or distantly metastatic melanoma.
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
- infusion, and
in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair or replacement of tissues for various conditions.