Kite Pharma files MAA at EMA for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) (NHL).

Kite Pharma, Inc. announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for autologous stem cell transplant. This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA.

Axicabtagene ciloleucel is currently under review by the FDA, and the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2017

The MAA for axicabtagene ciloleucel is supported by data from the ZUMA-1 trial, which met the primary endpoint of objective response rate (ORR), with 82 percent (p < 0.0001) of patients achieving a response after a single infusion of axicabtagene ciloleucel. 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 (CR). The most common Grade 3 or higher adverse events included cytokine release syndrome and neurologic events, which were generally reversible.

In May 2016, axicabtagene ciloleucel was granted access to its newly established Priority Medicines (PRIME) regulatory initiative for in the treatment of patients with refractory DLBCL. Access to the PRIME initiative is granted by the EMA to support the development and accelerate the review of new therapies to treat patients with a high unmet need..