FDA grants priority review to tisagenlecleucel for relapsed/refractory follicular lymphoma

On October 27, 2021, Novartis announced that the U.S. Food and Drug Administration (FDA) had accepted a supplemental biologics license application (sBLA) and granted priority review to tisagenlecleucel, a CD19-targeting chimeric antigen receptor (CAR) T-cell therapy, for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have received two prior lines of therapy. The European Medicines Agency (EMA) had also accepted Type II variation in the same patient population following orphan drug designation granted from the European Commission. If approved, this will be the third indication for tisagenlecleucel, a treatment option for patients with FL who relapse after standard therapies.^1,2

The FDA granted regenerative medicine advanced therapy (RMAT) designation to tisagenlecleucel for R/R FL in April 2020.

This priority review was granted based on positive data from the phase II ELARA trial (NCT03568461), a single-arm, multicenter study evaluating the efficacy and safety of tisagenlecleucel in patients with R/R FL. Updated results from the trial were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and reported on the Lymphoma Hub. ELARA enrolled 98 patients, and 97 patients received tisagenlecleucel. 94 were evaluable for efficacy; 79% of patients maintained a response ≥6 months, and among patients with a partial response (PR), 39% converted to a complete response (CR). At a median follow-up of 10.9 months, median progression-free survival (PFS), overall survival (OS), and duration of response (DoR) were not reached; the 6-month PFS was 76%. Any grade neurological adverse events (AEs) occurred in 9.3% of patients (1.0% Grade 3 or higher), and 48.5% of patients experienced any grade cytokine release syndrome (CRS); there were no reported Grade 3 or higher CRS events.³

Tisagenlecleucel is currently approved by the FDA, EMA, and other regulatory authorities for the treatment of R/R B-cell precursor acute lymphoblastic leukemia (B-ALL) in patients up to 25 years of age, and for the treatment of R/R diffuse large B-cell lymphoma (DLBCL) in adult patients after two or more lines of systemic therapy.^4,5