FDA grants breakthrough therapy designation to Novartis’s B-cell cancer

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Novartis’s CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

Novartis explained in its press release that CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. Also, during the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient’s cancer cells and other B-cells expressing a particular antigen.

“At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL,” said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis.

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

“We are encouraged by the FDA’s recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review,” said the Penn team’s leader, Carl June, MD, director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania.

Through the collaboration with Penn, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications. In March 2017, Novartis announced that the FDA accepted the company’s Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients, Novartis said.

The safety and efficacy profile of the study has not yet been established since CTL019 is an investigational therapy. Also, because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world, the company concluded.