Posted: Thursday, June 2, 2022
On May 27, the U.S. Food and Drug Administration (FDA) granted accelerated approval to tisagenlecleucel (Kymriah) for the treatment of adults with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. Tisagenlecleucel is a CD19-directed chimeric antigen receptor (CAR) T-cell therapy. It remains the only CAR T-cell therapy approved for both adults and children.
The agent’s approval for follicular lymphoma is based on data from the ELARA trial, a phase II, single-arm study. A total of 90 patients were evaluated in the primary efficacy analysis, with a median follow-up of about 17 months. During the initial study, 86% of patients treated with tisagenlecleucel achieved a response, with 68% experiencing a complete response. The median duration of response was not reached, with 75% of responders still in response at 9 months. For all 98 patients who underwent leukapheresis, the overall response rate was 86%, with a complete response rate of 67%.
For the 97 patients who were evaluable for safety at a median follow-up of 21 months, 53% experienced any-grade cytokine-release syndrome; however, there were no reported cases of cytokine-release syndrome of grade 3 or higher. A total of 43% of patients experienced any-grade neurologic events, with grade 3 or higher neurologic events seen in 6% of patients.