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The FDA has accepted for priority review the Biologics License Application for axicabtagene ciloleucel. The filing acceptance is supported by positive data demonstrated with a single infusion of axicabtagene ciloleucel in the ZUMA-1 phase II trial in patients with refractory aggressive non-Hodgkin lymphoma.

FDA; axicabtagene ci... - Click to enlarge in new windowFDA; axicabtagene ciloleucel. FDA; axicabtagene ciloleucel

Axicabtagene ciloleucel is an investigational therapy in which a patient's T cells are engineered to express a chimeric antigen receptor to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells.


The ZUMA-1 phase II trial met the primary endpoint of objective response rate recorded after a single infusion of axicabtagene ciloleucel with 82 percent (p<0.0001). At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response.


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