FDA Approval Summary: Lisocabtagene Maraleucel for Relapsed or Refractory Follicular Lymphoma
Cette étude analyse les données de l'essai ayant conduit la "Food and Drug Administration" à autoriser l'utilisation du lisocabtagène maraleucel pour traiter les patients atteints d'un lymphome folliculaire réfractaire ou récidivant
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.
Clinical Cancer Research , résumé, 2025