The U.S. Food and Drug Administration (FDA) has accepted for review the biologics license application (BLA) seeking the approval of axicabtagene ciloleucel (KTE-C19) for the treatment of refractory aggressive non-Hodgkin’s lymphoma (NHL).
The application was granted priority review status, which will reduce the review time from 10 months to six months. The FDA set an action date of Nov. 29, 2017.
“Patients with refractory aggressive NHL face a dire prognosis with only a 50 percent chance of surviving six months. This underscores the urgent medical need for these patients and why every day matters, from development to manufacturing to clinical experience,” David Chang, MD and PhD, executive vice president of research and development of Kite Pharma, said in a press release.
“We firmly believe in the potential for axicabtagene ciloleucel to address this need and forge a new path for the future of cell therapy,” said Chang, also chief medical officer of Kite.
Axicabtagene ciloleucel is a CAR (chimeric antigen receptor) T-cell therapy that removes the patient’s own T-cells and genetically engineers them to express a receptor that recognizes the CD19 protein, found at the surface of most lymphoma cells. The engineered cells are expanded in the lab and re-infused back into the patient’s bloodstream.
The FDA’s acceptance of the BLA review is supported by data from the ZUMA-1 Phase 2 trial (NCT02348216), an open-label, multi-center study assessing the safety and effectiveness of axicabtagene ciloleucel in 101 patients with refractory aggressive NHL.
The study, supported by the Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program, included one group of patients with diffuse large B-cell lymphoma, and another with transformed follicular lymphoma or primary mediastinal B-cell lymphoma patients.
ZUMA-1 met its primary endpoint of objective response rate after a single infusion of axicabtagene ciloleucel, with 82 percent of patients responding to the treatment, including 54 percent complete responses.
After a median follow-up of 8.7 months, 44 percent of patients had ongoing responses, including 39 percent complete responses. While the median overall survival had not been reached, the data compared favorably to the 6.6 months overall survival seen in a similar patient population treated with standard treatment regimens in the retrospective SCHOLAR-1 trial.
The most common grade 3 or higher adverse events included low levels of certain blood cell subsets, such as red blood cells, neutrophils, lymphocytes, and platelets. There were three deaths not due to disease progression, two of which were deemed related to axicabtagene ciloleucel.
Kite expects to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) requesting the approval of axicabtagene ciloleucel for the same indication.