Tecartus (brexucabtagene autoleucel), a cell-based gene therapy, received FDA approval for treatment of adult patients diagnosed with mantle cell lymphoma who have not responded to or who have relapsed following other kinds of treatment.
Tecartus is sponsored by Kite Pharma Inc. Tecartus, a CAR T-cell therapy, is the first cell-based gene therapy approved by FDA for the treatment of MCL.
MCL is a rare form of cancerous B-cell non-Hodgkin’s lymphoma that usually occurs in middle-aged or older adults. In patients with MCL, B-cells, a type of white blood cell which helps the body fight infection, change into cancer cells that start to form tumors in the lymph nodes and quickly spread to other areas of the body.
Approval was based on ZUMA-2 (NCT02601313), an open-label, multicenter, single-arm trial of 74 patients with relapsed or refractory MCL who had previously received anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody, and a Bruton tyrosine kinase inhibitor. Patients received a single infusion of brexucabtagene autoleucel following completion of lymphodepleting chemotherapy. The primary efficacy outcome measure was objective response rate per Lugano [2014] criteria as assessed by an independent review committee.
Of the 60 patients evaluable for efficacy based on a minimum duration of follow-up for response of six months, the ORR was 87% (95% CI: 75, 94), with a complete remission (CR) rate of 62% (95% CI: 48, 74). The estimated median duration of response was not reached (range of 0+ to 29.2+ months) after a median follow-up time for duration of response of 8.6 months. Of all 74 leukapheresed patients, the ORR as assessed by an independent review committee was 80% (95% CI: 69, 88) with a CR rate of 55% (95% CI: 43, 67).
Tecartus is being approved with a risk evaluation and mitigation strategy, which includes elements to assure safe use. The risk mitigation measures for Tecartus are identical to those of the current REMS Program for another CAR-T therapy, Yescarta.
To further evaluate the long-term safety of Tecartus, FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Tecartus.
Tecartus was approved under the Accelerated Approval pathway and was granted Priority Review and Breakthrough Therapy designations. Tecartus also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
