FDA Approves First CAR T-Cell Therapy for Marginal Zone Lymphoma, Offering New Hope for Patients

The U.S. Food and Drug Administration has announced the approval of Breyanzi (Lisocabtagene maraleucel) as the first Chimeric Antigen Receptor (CAR) T-cell therapy in the United States for adults diagnosed with marginal zone lymphoma (MZL). This groundbreaking treatment is indicated for patients who have not responded to or whose cancer has returned after at least two prior lines of therapy. CAR T-cell therapy represents a significant advancement in precision medicine, designed to genetically modify a patient’s own immune T-cells to more effectively identify and destroy cancer cells.

Marginal zone lymphoma is a rare and slow-growing cancer affecting the lymphatic system. It accounts for approximately 7% of all B-cell non-Hodgkin lymphoma cases, with an estimated 7,460 new diagnoses annually in the U.S. Patients with MZL who have not achieved a response or whose disease has relapsed after initial treatments, including those who have undergone hematopoietic stem cell transplant, often face a poorer prognosis. The approval of Breyanzi offers a new therapeutic avenue for these individuals.

The FDA’s decision was based on the evaluation of an open-label, multicenter, single-arm clinical trial. This study enrolled adults with relapsed or refractory MZL who had previously undergone at least two prior systemic therapies or had relapsed post-hematopoietic stem cell transplant. The trial involved a process called leukapheresis, where a patient’s own immune cells were collected for the manufacturing of Breyanzi. Following leukapheresis, patients received a single dose of Breyanzi after preparatory chemotherapy, intended to reduce the number of lymphocytes. Of the 77 patients who underwent leukapheresis, 66 received the single infusion of Breyanzi. The study demonstrated a high response rate, with 95.5% of patients experiencing a response to the treatment, and a complete response—meaning no detectable signs of marginal zone lymphoma on imaging scans—achieved by 62.1% of patients. These responses were observed to be durable, with a median follow-up of 21.6 months.

The most frequently reported adverse reactions associated with Breyanzi include cytokine release syndrome (CRS), diarrhea, fatigue, musculoskeletal pain, and headache. Comprehensive prescribing information for Breyanzi is available. The FDA granted Breyanzi Priority Review and Orphan Drug designation for this specific application. Traditional approval of Breyanzi for this indication was granted to Juno Therapeutics, Inc.

Article by Mel Anara, based upon information from the U.S. Food and Drug Administration.

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