FDA Approves First CAR T-Cell Therapy for Marginal Zone Lymphoma In the US
For Immediate Release:
December 04, 2025
The U.S. Food and Drug Administration today approved a new indication for Breyanzi (Lisocabtagene maraleucel) as the first Chimeric Antigen Receptor (CAR) T-cell therapy in the U.S. for treatment of adults with marginal zone lymphoma (MZL) who have failed treatment with or relapsed after two or more prior lines of therapy. Breyanzi is a chimeric antigen receptor CAR T-cell therapy that genetically engineers a patient’s own T-cells to target and kill cancer cells.
“Today’s approval represents a major advancement in precision medicine, essentially turning the patient’s immune system into a more effective tool to treat MZL,” Vinay Prasad, M.D., M.P.H., Chief Medical and Scientific Officer and Director of the FDA’s Center for Biologics Evaluation and Research. “The FDA continues to optimize its regulatory authority to expand treatment options in the fight against cancer.”
Marginal zone lymphoma is a rare slow-growing cancer of the lymphatic system. It presents about 7% of all B-cell non-Hodgkin lymphoma, with approximately 7,460 new cases occurring every year in the U.S. population. Patients with MZL who failed treatment or relapsed following treatment with upfront therapy have decreased survival.
“Today’s approval reflects the FDA’s continuing commitment to advance cell therapies to meet the needs of patients with rare cancers,” said Vijay Kumar M.D., Acting Director, Office of Therapeutic Products in the FDA’s Center for Biologics Evaluation and Research. “In granting approval, FDA took into consideration the high and durable response rate following a one-time treatment in patients with MZL, almost half of whom had either progression within two years of diagnosis or had refractory disease.”
The safety and effectiveness of Breyanzi were evaluated in an open-label, multicenter, single-arm trial in adults with relapsed or refractory MZL who had received at least two or more lines of systemic therapy or who relapsed after hematopoietic stem cell transplant (HSCT). Study participants underwent leukapheresis, a process to collect a patient’s own immune cells to manufacture Breyanzi. Patients received a single dose of Breyanzi two to seven days following the completion of preparatory chemotherapy to deplete recipient’s lymphocytes, a type of white blood cells. Among the 77 patients who underwent leukapheresis, 66 patients received the study specified single infusion of Breyanzi, of which 95.5% of patients experienced a response to treatment and 62.1% of patients had complete response without signs of marginal zone lymphoma on imaging scans. The responses were durable after a median follow up of 21.6 months.
The most common adverse reactions were cytokine release syndrome (CRS), diarrhea, fatigue, musculoskeletal pain, and headache. The full prescribing information for Breyanzi will be available here.
The FDA granted Breyanzi Priority Review and Orphan Drug designation for this application.
The FDA granted traditional approval of Breyanzi to Juno Therapeutics, Inc.
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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, radiation-emitting electronic products, and for regulating tobacco products.
Source: U.S. Food and Drug Administration (FDA)
