First Cell-Based Gene Therapy Approved For Treatment of Relapsed/Refractory MCL


Brexucabtagene autoleucel (Tecartus, Kite Pharma) is indicated for adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment.

Officials with the FDA have approved brexucabtagene autoleucel (Tecartus, Kite Pharma), a cell-based gene therapy for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. A chimeric antigen receptor (CAR) T cell therapy, brexucabtagene autoleucel is the first cell-based gene therapy approved by the FDA for the treatment of MCL, according to the agency.1

MCL is a rare form of cancerous B-cell non-Hodgkin lymphoma that typically occurs in adults who are middle-aged or older. 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.1

Each dose of brexucabtagene autoleucel is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T cells are collected and genetically modified to include a new gene that facilitates the targeting and killing of the lymphoma cells. These modified T cells are then infused back into the patient, according to the FDA.1

“Despite promising advances, there are still major gaps in treatment for patients with MCL who progress following initial therapy,” said Michael Wang, MD, lead investigator on the ZUMA-2 clinical trial, and professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, in a prepared statement. “Many patients have high-risk disease and are more likely to keep progressing, even after subsequent treatments.”2

The safety and efficacy of brexucabtagene autoleucel was established in the multicenter ZUMA-2 clinical trial of 60 adults with refractory or relapsed MCL who were followed for at least 6 months after their first objective disease response. The complete remission rate after treatment with the drug was 62%, with an objective response rate of 87%.1,2

The label for brexucabtagene autoleucel carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and for neurologic toxicities. Both CRS and neurologic toxicities can be fatal or life-threatening. The most common adverse effects of brexucabtagene autoleucel include serious infections, low blood cell counts, and a weakened immune system. Adverse effects from treatment usually appear within the first 1 to 2 weeks after treatment, but some adverse effects may occur later.1,2

Because of the risk of CRS and neurological toxicities, brexucabtagene autoleucel is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The risk mitigation measures for brexucabtagene autoleucel are identical to those of the current REMS Program for another CAR-T therapy from Kite Pharma, axicabtagene ciloleucel (Yescarta).­­­1,2

To further evaluate the long-term safety of brexucabtagene autoleucel, the FDA also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with the therapy.1

Brexucabtagene autoleucel was approved under the Accelerated Approval pathway. The drug previously was granted Priority Review and Breakthrough Therapy designations, and had received Orphan Drug designation. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.1

Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said the agency is seeing continued advances in the field of gene therapy, and is committed to supporting innovation in this new area of medicine. “Tremendous progress has been made in the discovery of new therapies for debilitating diseases that are difficult to treat,” said Marks, in a prepared statement.1


  • FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL [news release]. Silver Spring, MD; July 24, 2020: FDA website. Accessed July 24, 2020.
  • U.S. FDA Approves Kite’s Tecartus™, the First and Only CAR T Treatment for Relapsed or Refractory Mantle Cell Lymphoma [news release]. Santa Monica, CA; July 24, 2020: Kite Pharma website. Accessed July 24, 2020.

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