FDA Approves Liso-Cel for Relapsed/Refractory Marginal Zone Lymphoma

The FDA has approved lisocabtagene maraleucel (liso-cel; Breyanzi) for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy.

The regulatory decision was supported by findings from the MZL cohort of the phase 2 TRANSCEND FL trial (NCT04245839), which showed that patients in the intention-to-treat population achieved an overall response rate (ORR) of 84.4% (95% CI, 74.4%-91.7%), including a complete response (CR) rate of 55.8% (95% CI, 44.1%-67.2%). The median duration of response (DOR) was not reached (NR; 95% CI, 25.59-NR).

How was the TRANSCEND FL trial designed?

The phase 2 TRANSCEND FL trial was an open-label, global, multicenter study evaluating liso-cel in patients with relapsed or refractory B-cell non-Hodgkin lymphoma.2 Earlier findings from this study supported the May 2024 accelerated approval of liso-cel for relapsed or refractory follicular lymphoma after at least two prior lines of systemic therapy.3

Eligible patients were 18 years or older and had relapsed or refractory grade 1, 2, or 3a follicular lymphoma or relapsed/refractory MZL.2 All patients were required to have previously received at least one line of therapy that included an anti-CD20 antibody and an alkylating agent. For those with MZL, a minimum of 2 prior systemic therapies or relapse following hematopoietic stem cell transplant was required. Patients needed an ECOG performance status of 0 or 1 and adequate organ function.

Key exclusion criteria included a history or evidence of composite diffuse large B-cell lymphoma and follicular lymphoma, transformed follicular lymphoma, duodenal-type follicular lymphoma, or central nervous system–only disease. Prior CAR T-cell therapy or other genetically modified cellular therapies were not permitted.

Following leukapheresis, patients received lymphodepleting chemotherapy with fludarabine at 30 mg/m2 per day and cyclophosphamide at 300 mg/m2 per day for three days. Liso-cel was administered on day 1 at a target dose of 100 × 106 CAR-positive T cells.

The primary end point of the study was ORR, and secondary end points included CR rate, DOR, progression-free survival, overall survival, safety, and pharmacokinetics.

References

1. FDA approves lisocabtagene maraleucel for relapsed or refractory marginal zone lymphoma. FDA. December 4, 2025. Accessed December 4, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lisocabtagene-maraleucel-relapsed-or-refractory-marginal-zone-lymphoma
2. Bristol Myers Squibb presents first data from the marginal zone lymphoma cohort of the Transcend FL trial demonstrating deep and durable responses with Breyanzi (lisocabtagene maraleucel). News release. Bristol Myers Squibb. June 16, 2025. Accessed August 4, 2025. https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-First-Data-from-the-Marginal-Zone-Lymphoma-Cohort-of-the-Transcend-FL-Trial-Demonstrating-Deep-and-Durable-Responses-with-Breyanzi-lisocabtagene-maraleucel/default.aspx
3. FDA grants accelerated approval to lisocabtagene maraleucel for follicular lymphoma. FDA. May 15, 2024. Accessed December 4, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-lisocabtagene-maraleucel-follicular-lymphoma