U.S. Food and Drug Administration Approves First CAR-T Lymphoma Therapy: Axicabtagene Ciloleucel

New York, NY (October 19, 2017) – The U.S. Food and Drug Administration (FDA) announced it has approved the use of axicabtagene ciloleucel (YescartaTM, Kite, a Gilead Company) to treat patients with large B-cell lymphoma who have relapsed or have not responded to at least two previous lines of therapy. The second gene therapy approved by the FDA, this approval of the chimeric antigen receptor (CAR) T cell therapy, represents the first indication of gene therapy for lymphoma patients.

“With this first-of-its kind approval for a CAR-T cell therapy, we are entering a new era in the way we approach treatment of blood cancers,” said Meghan Gutierrez, Chief Executive Officer of the Lymphoma Research Foundation. “We applaud the leadership of the FDA, as well as the researchers, physicians, nurses and patients, who have helped to usher in this historic discovery, providing hope for certain lymphoma patients who previously had limited treatment options.”

CAR-T cell therapies provide engineered molecules called chimeric antigen receptors (CARs) that recognize and destroy antigens present on the surface of lymphoma cells, and are manufactured
individually for each patient. The FDA has approved this CAR-T cell therapy for use in diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma, or TFL) patients with relapsed / refractory disease after two previous treatments.

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” FDA Commissioner Scott Gottlieb, M.D. said in a statement. “This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”

The Lymphoma Research Foundation offers educational materials and resources on the subject of immunotherapy in the treatment of lymphoma, such as videos, webcasts, and fact sheets. These materials include information on CAR-T cell therapy and are available at no cost to lymphoma patients and their loved ones. In addition to these resources, the Lymphoma Research Foundation will host its annual North American Educational Forum on Lymphoma from October 20-22, 2017 at the New York Marriott at the Brooklyn Bridge, in which Dr. Bruce Cheson, MD, FACP, FAAAS, Georgetown University Hospital Lombardi Comprehensive Cancer Center, Past Chair of LRF’s Scientific Advisory Board and Dr. Stephen J. Schuster, MD, Hospital of the University of Pennsylvania will host a dedicated educational lunch program on Immune Oncology. During this session, patients can learn more about immunotherapy and this new approval of CAR-T cell therapy and ask questions of leading experts in the field. To register for the 2017 North American Educational Forum on Lymphoma, visit www.lymphoma.org/EdForum.

For more on the FDA approval of CAR-T cell therapy, click here. For additional information on diffuse large B-cell lymphoma, visit www.focusondlbcl.org, and sign up to learn more at www.lymphoma.org for upcoming updates on CAR-T-specific resources.

About the Lymphoma Research Foundation
The Lymphoma Research Foundation (LRF) is the nation’s largest non-profit organization devoted to funding innovative research and serving the lymphoma community through a comprehensive series of education programs, outreach initiatives and patient services. To date, LRF has awarded nearly $60 million in lymphoma-specific research.

For additional information on LRF’s research, education and services, visit lymphoma.org.

Jake DiGregorio