Author: Mark Dresser, PhD on October 10, 2019
In a recent Commentary published in Clinical and Translational Science (CTS), Dr. Peter Marks, the Director of the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER), provides a clear and compelling overview of the FDA's flexible regulatory approach to the evaluation and review of chimeric antigen receptor-T (CAR-T) cell therapies. CAR‐T cells have produced remarkable clinical results in the treatment of a number of hematologic malignancies, and they are currently under investigation to treat many other disorders such as solid tumors and autoimmune and infectious diseases. Given the tremendous promise of this emerging therapeutic approach, there has been a corresponding surge in the research and development of new CAR-T cell therapies. To date, two CAR-T cell therapies are approved in the US - Tisagenlecleucel (Kymriah) and Axicabtagene ciloleucel (Yescarta) - for the treatment of hematologic malignancies, and there are more than 100 active investigational new drug (IND) applications in the United States alone.
Dr. Marks's Commentary summarizes the FDA's efforts to assist product developers of CAR-T cell therapies. For example, CBER has recently issued numerous gene therapy guidance documents on topics ranging from Regulatory Considerations for Human Cell, Tissues, and Cellular and Tissue‐Based Products: Minimal Manipulation and Homologous Use to Long Term Follow‐up After Administration of Human Gene Therapy Products. In addition, CBER has also implemented the Regenerative Medicine Advanced Therapy (RMAT) designation program which has all of the benefits of the Breakthrough Therapy (BT) designation. As of May 1, 2019, 100 requests and 34 products have been granted RMAT designation. In his Commentary, Dr. Marks discusses the rationale for CBER's decisions on whether to accept or decline RMAT designation applications. Dr Marks also discusses the benefits to sponsors who take advantage of CBER's INitial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT) program at very early stages of development of a product.
This Commentary will be of great interest to researchers and developers of new CAR-T cell therapies. The CTS Editorial Team would like to thank Dr. Marks for his outstanding contribution to Clinical and Translational Science. We welcome the submission of original research articles on this promising new treatment paradigm.
Image by Marks. Clin. Trans. Sci., https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.12666, is licensed under CC BY-NC 4.0. Published 2019. This article is a U.S. Government work and is in the public domain in the USA. Clinical and Translational Science published by Wiley Periodicals, Inc. on behalf of the American Society for Clinical Pharmacology and Therapeutics.
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