CTL019 gets another set of FDA wings.

Novartis’ treatment for adult patients with relapsed and refractory diffuse large B-cell lymphoma was granted Breakthrough Therapy designation by the FDA. The approval covers CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy for patients who have failed two or more prior therapies.

Rather than infusing or ingesting a compound to achieve therapeutic effect, during the Personalized CAR-T therapy treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create enhanced T cells. These enhanced cells are genetically coded to hunt cancer cells and other B-cells expressing a particular antigen. This is the second indication for CTL019; the first being granted in March for the treatment of r/r B-cell acute lymphoblastic leukemia in pediatric and young adult patients. It is also the 14th Breakthrough Therapy designation Novartis has received since the FDA initiated the program in 2013.

Novartis’ Chief Medical Officer and Global Head of Drug Development, Vas Narasimhan, spoke on the FDA’s decision: “We are eager to unlock the full potential of CTL019, including the potential to help patients with relapsed and refractory diffuse large B-cell lymphoma [and] look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible." FDA guidance notes therapeutic agents that receive Breakthrough Therapy designation treat serious and life threatening diseases and demonstrate higher performance over existing therapies (on one or more clinically significant endpoints) based on preliminary clinical evidence.

First developed by the University of Pennsylvania in 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies for the investigational treatment of cancers, including CTL019.