Phase II study of tisagenlecleucel in patients with R/R FL meets its primary endpoint

On August 4, 2020, positive results were announced from the phase II ELARA trial (NCT03568461), investigating tisagenlecleucel in patients with relapsed or refractory (R/R) follicular lymphoma (FL).

Tisagenlecleucel is an anti‐CD19 chimeric antigen receptor T (CAR T) cell, currently approved for the treatment of pediatric and young adult patients (up to 25 years of age) with R/R acute lymphoblastic leukemia, and adult patients with R/R  diffuse large B-cell lymphoma. In April, tisagenlecleucel received U.S. Food and Drug Administration (FDA) regenerative medicine advanced therapy designation for the treatment of patients with R/R FL, based on preliminary results from the ELARA trial.

The ELARA trial is a single-arm, multicenter, open-label trial investigating the efficacy and safety of tisagenlecleucel in adult patients with R/R FL. At the interim analysis, the study met the primary endpoint of complete response rate, with no new safety signals observed.

Severe side effects associated with tisagenlecleucel included cytokine release syndrome (CRS) and neurological toxicities. Due to the risk of CRS and neurologic toxicities, tisagenlecleucel is only available through a restricted program under a risk evaluation and mitigation strategy.

Results from the ELARA trial will be included in US and EU regulatory submissions.