FDA Approves Lisocabtagene Maraleucel for the Treatment of Relapsed or Refractory Marginal Zone Lymp

On December 4, 2025, the U.S. Food and Drug Administration (FDA) approved lisocabtagene maraleucel (Breyanzi, developed by Juno Therapeutics, a subsidiary of Bristol Myers Squibb) 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.

About the TRANSCEND FL-MZL Cohort Study

Efficacy was evaluated based on the TRANSCEND FL-MZL cohort study (ClinicalTrials.gov Identifier: NCT04245839), an open-label, multicenter, single-arm clinical trial of adult patients with relapsed or refractory MZL who had received at least two or more prior lines of systemic therapy or relapsed after hematopoietic stem cell transplantation (HSCT). Patients were required to have an Eastern Cooperative Oncology Group (ECOG) performance status of 1 or lower.

Patients received a single dose of lisocabtagene maraleucel infusion on Days 2 to 7 following lymphodepleting chemotherapy (fludarabine 30 mg/m²/day plus cyclophosphamide 300 mg/m²/day, administered concurrently for 3 days). Efficacy analyses were based on 77 patients who underwent leukapheresis (intent-to-treat [ITT] population) and 66 treated patients who met the following criteria: presence of measurable disease confirmed by computed tomography (CT) at baseline, receipt of a qualifying product within the target dose range, and at least 9 months of follow-up from the first response.

The primary efficacy endpoints were overall response rate (ORR; percentage of patients achieving a best overall response of complete response [CR] or partial response [PR] per Lugano Criteria) and duration of response (DOR) as assessed by an independent review committee (IRC). In the ITT population, the ORR was 84.4% (95% confidence interval [CI]: 74.4, 91.7), and the CR rate was 55.8% (95% CI: 44.1, 67.2). The median DOR was not reached (95% CI: 25.59, not reached).

About Dosage and Administration

The recommended dose of lisocabtagene maraleucel is 90 to 110 × 10⁶ CAR-positive viable T cells, with a CD4:CD8 component ratio of 1:1.

This application was granted Priority Review. Lisocabtagene maraleucel previously received Orphan Drug Designation. FDA’s accelerated programs for drugs and biologics for serious conditions are described in the Industry Guidance: Accelerated Programs for Drugs and Biologics for Serious Conditions. FDA’s accelerated programs for regenerative medicine advanced therapies (RMAT) are detailed in the Industry Guidance: Accelerated Programs for Regenerative Medicine Therapies for Serious Conditions.