FDA newly approves Lisocabtagene maraleucel to treat relapsed or refractory marginal zone lymphoma

On December 4, 2025, the Food and Drug Administration (FDA) approved Lisocabtagene maraleucel for the treatment of adults with relapsed or refractory marginal zone lymphoma (MZL; non-Hodgkin lymphoma). The approval, which applies to patients who have received at least two prior systemic therapies, marks the new therapy as a new treatment option for patients with refractory lymphoma.

The efficacy of lisocabetagen was evaluated in theTRANSCEND FL-MZL cohort (NCT04245839), an open-label, multicenter, single-arm study in adults with relapsed or refractory MZL. Participants were required to have relapsed after at least two systemic therapies or after hematopoietic stem cell transplantation (HSCT) and had an ECOG score of 1 or less. Patients received a single dose of lisocabeta root with fludarabine 30 mg/m²/day and cyclophosphamide 300 mg/m²/day for three days 2 to 7 days after completion of lymphodepleting chemotherapy.

The study conducted an efficacy analysis on77 intention-to-treat (ITT) patients and 66 treated patients. All patients had measurable disease confirmed by computed tomography (CT) scan at baseline and received qualifying product within the expected dose range, with follow-up for at least 9 months. The primary efficacy outcome measures were overall response rate (ORR), defined as the percentage of patients in complete response (CR) or partial response (PR), and duration of response (DOR) as determined by an independent review committee (IRC).

The study results showed thatThe ORR of the ITT population was 84.4% (95% confidence interval: 74.4% to 91.7%), of which the CR rate was 55.8% (95% confidence interval: 44.1% to 67.2%), which did not reach the median risk ratio (NR) (95% confidence interval: 25.59, NR).

The recommended dose of lisocabeta root is 90 to 110×10^6 CAR-positive live T cells, and the ratio of CD4 to CD8 cells is 1:1. This approval provides new hope for patients with relapsed or refractory MZL and further advances the development of personalized cancer treatments.

The prescribing information lists warnings and precautions for the use of lisocabeta root, including cytokine release syndrome (CRS), neurotoxicity, allergic reactions, severe infections, long-term cytopenias, hypogammaglobulinemia, secondary malignancies, and immune effector cell-related hemophagocytic lymphohistiocytosis-like syndromes.

References:https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lisocabtagene-maraleucel-relapsed-or-refractory-marginal-zone-lymphoma