Based on the results of the phase 2 ZUMA-1 trial in patients with refractory non-Hodgkin lymphoma, Kite Pharma has submitted for, and received, a priority review for its chimeric antigen receptor (CAR)-T cell treatment, axicabtagene ciloleucel.
Based on the results of the phase 2 ZUMA-1 trial in patients with refractory non-Hodgkin lymphoma (NHL), Kite Pharma has submitted for, and received, a priority review from the FDA for its chimeric antigen receptor (CAR)-T cell treatment, axicabtagene ciloleucel. This will slice off 4 months from the review process, from 10 months to 6.
The submission comes within a few months of the company announcing positive results in patients with chemotherapy-resistant aggressive B-cell NHL who were administered a single infusion of axicabtagene ciloleucel. The trial met its primary endpoint of objective response rate (ORR), with 82% of patients achieving an ORR (P <.0001); 44% of patients had an ongoing response at 8.7 months, 39% of whom had a complete response. Updated trial results will be presented on June 5th at the annual meeting of the American Society of Clinical Oncology in Chicago, Illinois.
“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,” said David Chang, MD, PhD, executive vice president of research and development and chief medical officer of Kite. “We firmly believe in the potential for axicabtagene ciloleucel to address this need and forge a new path for the future of cell therapy.”
A variety of hematological adverse events were documented (eg, anemia, neutropenia, thrombocytopenia, etc) in patients administered axicabtagene ciloleucel, and a patient death was reported just earlier this month, due to cerebral edema. This patient, according to the company, had inadequate responses to 2 lines of therapy and had rapidly progressive and symptomatic disease when enrolled. On a conference call with investors, Chang said, “The explosive nature of the patient’s lymphoma immediately prior to and up to the day of axi-cil therapy is a consideration and is being further investigated.”
Another company, Juno Therapeutics, had to halt its CAR-T ROCKET trial earlier this year following 5 patient deaths in patients with acute lymphoblastic leukemia. This will be a significant consideration for the FDA as it reviews the data from ZUMA-1.
The Prescription Drug User Fee Act target action date for axicabtagene ciloleucel is November 29, 2017.