Kite, a Gilead Company announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion on the company’s Marketing Authorization Application (MAA) for Yescarta (axicabtagene ciloleucel) as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy.
Axicabtagene ciloleucel is a chimeric antigen receptor T cell (CAR T) therapy that modifies a patient’s own T cells to recognize and attack cancer cells and has the potential to induce complete responses (no detectable cancer) in a proportion of patients with aggressive forms of non-Hodgkin lymphoma (NHL). Axicabtagene ciloleucel was granted PRIME status by the EMA in May 2016.
The CHMP positive opinion was adopted following review by European Union (EU) regulators, including the Committee for Advanced Therapies and the CHMP. The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for use in the 28 countries of the EU, Norway, Iceland and Liechtenstein.
“This CHMP positive opinion is an important milestone for those patients in the European Union living with DLBCL or PMBCL,” said Alessandro Riva, MD, Gilead’s Executive Vice President, Oncology Therapeutics & Head, Cell Therapy. “The recommendation brings axicabtagene ciloleucel one step closer to adult patients who currently have few or no treatment options available to them and we are focused on providing access to this innovative treatment as quickly as possible.”
The MAA is supported by data from the ZUMA-1 trial of axicabtagene ciloleucel in adult patients with refractory aggressive NHL. In the single-arm trial, 72 percent of patients (n=73/101) who received a single infusion of axicabtagene ciloleucel responded to therapy with 51 percent (n=52/101) achieving a complete response (as assessed by an independent review committee, median follow up of 15.1 months). Grade 3 or higher adverse events reported during ZUMA-1 included, but were not restricted to, cytokine release syndrome (CRS), neurologic events and cytopenias.
DLBCL is the most common form of NHL and represents an area of significant unmet need. The prognosis for patients with refractory DLBCL is very poor, with a median survival of just six months. Across the EU in 2018, there are an estimated 7,700 patients with DLBCL who are refractory to, or have relapsed after, two or more lines of therapy, and may be eligible for CAR T therapy.
Axicabtagene ciloleucel was approved by the U.S. Food and Drug Administration on October 18, 2017. In the EU, axicabtagene ciloleucel is an investigational product and its efficacy and safety have not been established.