On July 24, 2020, the FDA accelerated the approval of brexucabtagene autoleucel (Tecartus; Kite Pharma), a CAR T-cell therapy, for the treatment of adults with mantle-cell lymphoma (MCL) whose disease has not responded to or has relapsed after other therapies. Brexucabtagene autoleucel is the first gene therapy FDA-approved specifically for the treatment of patients with MCL, a rare type of B-cell non-Hodgkin’s lymphoma.
The FDA granted brexucabtagene autoleucel a priority review, a breakthrough therapy designation, and an orphan drug designation.
Commenting on this FDA approval, Peter Marks, MD, PhD, Director of the FDA’s Center for Biologics Evaluation and Research, said, “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. We’re seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.”
The FDA approved brexucabtagene autoleucel based on safety and efficacy results from a multicenter clinical trial of 60 adults with relapsed or refractory MCL who were followed for ≥6 months after their first objective response to treatment. The complete remission rate after treatment with brexucabtagene autoleucel was 62%, with an objective response rate of 87%.
Brexucabtagene autoleucel is created from the patient’s own immune system by collecting the patient’s cells and genetically modifying them to target the lymphoma cells. These modified T-cells are then infused back into the patient.
The most common (≥20%) adverse reactions occurring with brexucabtagene autoleucel include serious infections, low blood cell counts, and a weakened immune system. Side effects from treatment usually appear within the first 1 to 2 weeks after treatment, but some side effects may occur later.
Furthermore, brexucabtagene autoleucel is associated with a risk for cytokine release syndrome, a life-threatening condition, and neurologic toxicities. Because of these serious risks, brexucabtagene autoleucel is available only through a Risk Evaluation and Mitigation Strategy (REMS) program. This REMS program is identical to that of another CAR-T therapy (axicabtagene ciloleucel; Yescarta) from the same manufacturer.
To evaluate the long-term safety of brexucabtagene autoleucel, the FDA is requiring the manufacturer to conduct a postmarketing observational study of patients who received treatment with brexucabtagene autoleucel.