New CAR T-Cell Therapy for Non-Hodgkin Lymphoma Sent to FDA

A biologics license application has been submitted to the U.S. Food and Drug Administration (FDA) for axicabtagene ciloleucel (formerly known as KTE-C19) for the treatment of patients with aggressive non-Hodgkin lymphoma (NHL) who cannot undergo autologous hematopoietic cell transplantation (AHCT).

Axicabtagene ciloleucel is an investigational chimeric antigen receptor (CAR) therapy that targets the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias.

The application was supported by data from the ZUMA-1 trial, in which an objective response rate of 83 percent (primary endpoint) and a complete response rate of 54 percent were achieved after a single treatment infusion (p<0.0001). In ZUMA-1, 101 patients were treated with axicabtagene ciloleucel. Patients had either diffuse large B-cell lymphoma (n=77), primary mediastinal B-cell lymphoma, or transformed follicular lymphoma (latter two combined for n=24). Most patients had stage III/IV disease (85%) and were refractory to chemotherapy and had no prior AHCT (79%). About one-fifth of patients had relapsed within 12 months of AHCT (21%). Patients were heavily pretreated, with 69 percent having received three or more lines of prior therapy.

The most common grade 3 or higher adverse events were: anemia (43%), neutropenia (39%), decreased neutrophil count (32%), febrile neutropenia (31%), leukopenia (29%), thrombocytopenia (24%), encephalopathy (21%), and decreased lymphocyte count (20%).

Axicabtagene ciloleucel was granted breakthrough therapy designation by the FDA for diffuse large B-cell lymphoma in December 2015.

Source: Kite Pharma press release, April 2, 2017; Kite Pharma press release March 31, 2017