Kite Pharma submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) requesting the approval of its CAR T-cell therapy, axicabtagene ciloleucel, as treatment for patients with certain lymphomas.
The include relapsed or refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL), who cannot receive autologous stem cell transplants.
This is the first application for a CAR T-cell therapy ever submitted to the EMA.
“The MAA submission of axicabtagene ciloleucel marks an important global milestone in the development of engineered T-cell therapy,” Arie Belldegrun, MD, president and CEO of Kite, said in a press release.
He said the company is excited to be working with the EMA’s Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) “to help bring this potentially transformative therapy to patients in the EU.”
Axicabtagene ciloleucel, formerly known as KTE-C19, is a CAR (chimeric antigen receptor) T-cell therapy. The treatment consist of collecting the patient’s own T-cells and modifying them to express a CAR protein that recognizes the surface protein CD19, a molecule that is widely expressed by B-cell lymphomas and leukemias.
Kite’s application includes primary data from the ZUMA-1 trial (NCT02348216), a Phase 1/2 trial testing axicabtagene ciloleucel in treatment-resistant or relapsed aggressive non-Hodgkin’s lymphoma patients. The trial included 101 patients with DLBCL, PMBCL, or TFL, most with advanced-stage disease.
Data presented late June at the 22nd Congress of the European Hematology Association (EHA), showed that 82 percent of patients responded to treatment after a single infusion of axicabtagene ciloleucel. This positive response was sustained in 44 percent of patients after a median follow-up time of 8.7 months. At that time, 39 percent of patients exhibited a complete response.
The most common severe treatment-related adverse events reported in ZUMA-1 included cytokine release syndrome and neurologic events, which were generally reversible after suitable management.
Axicabtagene ciloleucel has been designated a breakthrough therapy by the U.S. Food and Drug Administration for the treatment of DLBCL, TFL, and PMBCL, and received priority medicines (PRIME) regulatory support in the European Union. These designations are meant to support a drug’s development and accelerate its regulatory review.
The FDA is reviewing Kite’s biologics license application (BLA) submitted for axicabtagene ciloleucel for the treatment of refractory aggressive non-Hodgkin’s lymphoma. A final decision is expected by Nov. 29.