Kite Pharmaceutical's marketing authorization application, or MAA, is the first to ever be submitted to the European Medicines Agency, or EMA.
Arie Belldegrun, MD, FACS, Chairman President, and Chief Executive Officer of Kite stated, "the MAA submission of axicabtagene ciloleucel marks an important global milestone in the development of engineered T cell therapy. We are excited to work closely with the EMA, 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."
The ZUMA-1 trial is one of the largest supporting factors in Kite Pharmaceutical's MAA for axicabtagene ciloleucel. ZUMA-1 is a phase 1/2 trial that has tested axicabtagene ciloleucel in non-Hodgkin's lymphoma patients that have shown to be either treatment-resistant or relapsed aggressive. Specifically, the trial analyzed 101 patients who had either DLBCL, PMBCL, or TFL. Most of these patients were had advanced-stage diseases.
The data from the trial showed that after just one infusion of axicabtagene ciloleucel, 82 percent of patients displayed a response. This result meets the primary endpoint of objective response rate, or the ORR. After over eight months, 44 percent of patients were displaying an ongoing response, including the 39 percent of these patients who exhibited a complete response.
However, not all results were positive. There were adverse effects of the treatment found, the most common of which was cytokine release syndrome and neurologic events. Although these are Grade 3 or higher effects that are largely unwanted in treatment, they were found to be reversible when managed properly.
The findings of the ZUMA-1 were presented at the 22nd Congress of the European Hematology Association, or EHA. These findings are the backbone of Kite Pharmaceutical's MAA.