FDA approval of CAR T-cell therapies for hematologic cancers. It was manufactured by the Novartis Pharmaceuticals corporation. The CAR T-cell therapy in question is tisagenlecleucel (Kymriah). Known for most of its development as CTL019, tisagenlecleucel was approved for the treatment of certain pediatric patients and young adults with acute lymphoblastic leukemia (ALL).
Specifically, it was approved to treat children and young adults up to 25 years of age with B-cell ALL that is refractory, meaning that it has not responded to standard treatments or has relapsed at least twice. CAR T-cell therapy, a type of immunotherapy, is an especially important breakthrough for children with leukemia and for adults with certain hard-to-treat types of blood cancer. It also shows promise for other diseases. While CD19 and BCMA are the only antigens for which there are FDA-approved CAR T cell therapies, CAR T cell therapies have been developed that target other antigens that are frequently found in blood cancers, including therapies that attack several antigens at the same time.
Tisagenlecleucel was the most expensive cancer treatment approved in the United States. Department of State, which, with good reason, raises questions about the cost-effectiveness of treatment.
