FDA Approves First Cell-Based Gene Therapy for MCL

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The therapy, indicated for a rare form of B-cell non-Hodgkin’s lymphoma, was developed by Kite, a Gilead company, which will manufacture the therapy at its facility in El Segundo, CA.

FDA approved Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for treating mantle cell lymphoma (MCL) from Kite, a Gilead company. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by FDA for MCL, the agency announced in a July 24, 2020 press release. The therapy is indicated for adult patients diagnosed with MCL who have not responded to, or who have relapsed following, other kinds of treatment.

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 change into cancer cells that start to form tumors in the lymph nodes and quickly spread to other areas of the body.

Tecartus is the individual patient’s own T cells that have been collected and genetically modified to include a new gene that facilitates the targeting and killing of the lymphoma cells. The therapy is administered via infusion back into the patient.

The safety and efficacy of Tecartus was established in a multicenter clinical trial of 60 adults with refractory or relapsed MCL who were followed for at least six months after their first objective disease response. FDA reports that the complete remission rate after treatment with Tecartus was 62%, with an objective response rate of 87%.

“Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat. This approval is yet another example of customized treatments that use a patient’s own immune system to help fight cancer, while using a scientific advance in this promising new area of medicine,” said Peter Marks, MD, PhD, director of FDA’s Center for Biologics Evaluation and Research, in the press release. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”


To further evaluate the long-term safety of Tecartus, FDA is also requiring Kite to conduct a post-marketing observational study involving patients treated with the therapy.

Tecartus will be manufactured in Kite’s commercial manufacturing facility in El Segundo, CA. In one of its clinical trials, Kite demonstrated a 96% manufacturing success rate and a median manufacturing turnaround time of 15 days from leukapheresis to product delivery. Manufacturing speed is especially critical for patients with advanced disease, who are very ill and at risk for quick progression.

Source: FDA and Kite