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News From the Food and Drug Administration
September 1, 2020

CAR-T Therapy Is Approved for Mantle Cell Lymphoma

JAMA. 2020;324(9):832. doi:10.1001/jama.2020.15456

The first cell-based gene therapy for adults with mantle cell lymphoma (MCL) has received FDA approval.

Brexucabtagene, marketed as Tecartus, is a chimeric antigen receptor T-cell (CAR-T) therapy indicated for patients who haven’t responded to other MCL treatments or have relapsed following therapy. CAR-T therapy involves removing a patient’s own T cells, genetically engineering them to recognize and destroy lymphoma cells, and then infusing them back into the patient.

Approval of the drug “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,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.

In a single-group clinical trial, 68 patients with refractory or relapsed mantle cell lymphoma received 1 infusion of brexucaptagene. Among 60 patients who were followed up for at least 6 months, 87% responded to the drug and 62% had a complete remission. Common adverse reactions included fever, hypotension, infections, encephalopathy, fatigue, tachycardia, and arrhythmia. Although most adverse effects appeared within 1 or 2 weeks after treatment, the FDA noted that some could occur later.

The drug’s label carries a boxed warning cautioning physicians and patients about the risk of developing potentially life-threating cytokine release syndrome and neurological toxicities. Because of those risks, brexucaptagene was approved with a risk evaluation and mitigation strategy to help ensure safe use. The FDA has also required the manufacturer, Kite Pharma Inc, of Santa Monica, California, to conduct a postmarketing observational study of patients who receive the drug. Its reported list price is $373 000.