FDA accepts filing and grants priority review for CAR-T therapy, CTL 019 (tisagenlecleucel-T) to treat B-cell acute lymphoblastic leukemia.-Novartis

Novartis announced that the FDA has accepted the company's Biologics License Application (BLA) filing and granted priority review for CTL 019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

CAR-T is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. 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.

The priority review designation and BLA submission for CTL019 is based on the results from the Novartis-sponsored ELIANA study (NCT02435849) , the first global CAR-T cell trial with study enrollment having occurred across 25 centers in the US, EU, Canada, Australia and Japan. In the Phase II study, 82% (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 CTL 019 infusion. The data were presented at the American Society of Hematology meeting in December 2016 (Abstract #221).

The submission is also supported by findings from a US multicenter trial and an earlier single site trial led by the Children's Hospital of Philadelphia (CHOP) examining the safety and efficacy of CTL 019 among pediatric and young adult patients with r/r B-cell ALL. Stephan Grupp, MD, PhD, from CHOP was the lead investigator of the trials.