For Immediate Release:

Today, the U.S. Food and Drug Administration approved Tecartus (brexucabtagene autoleucel), 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. Tecartus, a chimeric antigen receptor (CAR) T cell therapy, is the first cell-based gene therapy approved by the FDA for the treatment of MCL.

“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, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”

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 help 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.

Each dose of Tecartus is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T cells, a type of white blood cell, 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.

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. The complete remission rate after treatment with Tecartus was 62 percent, with an objective response rate of 87 percent.

The label 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 side effects of Tecartus include serious infections, low blood cell counts and a weakened immune system. Side effects from treatment usually appear within the first one to two weeks after treatment, but some side effects may occur later.

Because of the risk of CRS and neurological toxicities, Tecartus is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). 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, the 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. The Tecartus application was reviewed using a cross-agency approach. 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. The FDA remains committed to supporting safe and effective treatment options that have the potential of providing lifesaving results.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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