Novartis has stepped ahead in the race to get the first cancer-killing CAR-T cell therapy on the market having successfully filed a biologics license application with US regulators.
The company is seeking permission to market its investigational chimeric antigen receptor T cell (CAR-T) therapy CTL019 (tisagenlecleucel-T) for relapsed and refractory paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL).
The US Food and Administration has assigned the submission a priority review, which means that the therapy could be approved within just six months, potentially leap-frogging its closest rival - Kite’s axicabtagene ciloleucel (KTE-019) - to the finish line. Kite kicked off a rolling submission for its therapy back in December, and is reportedly expecting to complete its filing shortly.
CAR-T offers a new treatment approach in that it is specifically manufactured for each individual patient. During the 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.
Novartis’ filing contains data - presented at the American Society of Haematology meeting in December 2016 - from a Phase II study in which 82 percent (41 of 50) of patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery at three months post CTL019 infusion.
"Even if a patient has difficult-to-treat relapsed/refractory leukaemia, we have seen treatment with CTL019 in clinical trials put cancer into remission," said Grupp, director of the Cancer Immunotherapy Frontier Program and Director of Translational Research for the Center for Childhood Cancer Research at CHOP. "This could be a first-of-its-kind treatment with exciting potential to help pediatric and young adult r/r B-cell ALL patients."
Novartis picked up rights to CTL019 under an agreement with the University of Pennsylvania in 2012, which also gives it worldwide rights to CARs developed in all cancer indications.
“The past five years have seen tremendous progress in the development and application of cellular engineering in an effort to personalise the treatment of cancer," 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. "We now know that it is possible to treat patients in clinical trials across the world using this approach, and the results we have observed mark a potential new paradigm in the treatment of blood cancers that have not responded to standard therapies."
If successful, both Novartis’ and Kite’s CAR-T therapies are each expected to rake in billions of dollars in peak sales.