FDA Approval Summary: Lisocabtagene Maraleucel for Relapsed or Refractory Follicular Lymphoma.

In May 2024, the FDA granted accelerated approval to lisocabtagene maraleucel for the treatment of adults with relapsed or refractory (R/R) follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. The single-arm, open-label, multicenter, phase 2 TRANSCEND-FL trial evaluating a single dose of lisocabtagene maraleucel in adults with R/R indolent non-Hodgkin lymphoma supported the FL indication. The trial enrolled 139 patients with R/R FL, including 107 with two or more prior lines of therapy. Of the 107 patients, 94 patients comprised the primary efficacy population and the independent review committee-assessed overall response rate was 95.7% (95% confidence interval [CI]: 89.5, 98.8), with 73.4% (95% CI: 63.3, 82.0) achieving complete response. The median duration of response was not reached (95% CI: 18.0 months, NR) and the 1-year remission rate was 81% (95% CI: 71, 88). Serious adverse reactions occurred in 26% of patients, most often due to cytokine release syndrome (CRS), neurologic toxicity, and febrile neutropenia. CRS occurred in 59% (Grade 3: 1%) of patients and neurologic toxicities occurred in 15% (Grade 3: 2%). As a condition of accelerated approval, post marketing studies were required to verify the clinical benefit of lisocabtagene maraleucel in R/R FL. This article summarizes key aspects of the regulatory review.