FDA clears first cell-based gene therapy for lymphoma

FDA approved the first-cell-based gene therapy to treat mantle cell lymphoma (MCL).

Brexucabtagene autoleucel (Tecartus, Kite, a Gilead company), a chimeric antigen receptor (CAR) T cell therapy, received Priority Review and Breakthrough Therapy Designation by FDA.

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“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 the FDA’s Center for Biologics Evaluation and Research, in a 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.”

Each dose of Tecartus is a customized treatment created using a patient’s own immune system to help fight the lymphoma, FDA said. 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. The modified T cells are then infused back into the patient.

“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 of the Tecartus trial and professor at The University of Texas MD Anderson Cancer Center, in a Kite press release. “Many patients have high-risk disease and are more likely to keep progressing, even after subsequent treatments.

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“The availability of Tecartus as the first-ever cell therapy for patients with relapsed/refractory MCL provides an important option with a response rate of nearly 90% and early clinical evidence suggesting durable remissions in later lines of therapy,” Wang added.

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 6 months after their first objective disease response. The complete remission rate after treatment with Tecartus was 62% and the treatment boasted an objective response rate of 87%.

Tecartus’s label carries a boxed warning for cytokine release syndrome (CRS), 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.

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).

FDA is also requiring Kite to conduct a post-marketing observational study involving patients treated with Tecartus.

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