The US Food and Drug Administration (FDA) has granted approval to tisagenlecleucel for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma who have failed 2 or more systemic therapy regimens, according to a press release.1 Indications for tisagenlecleucel now include diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma, and follicular lymphoma–transformed DLBCL.

Tisagenlecleucel is the only FDA-approved chimeric antigen receptor (CAR)-T cell therapy indicated for non-Hodgkin lymphomas (NHL) and B-cell acute lymphocytic leukemia (ALL). 

The FDA based its approval on results of the single-arm phase 2 JULIET clinical trial ( Identifier: NCT02445248), in which researchers enrolled patients with R/R DLBCL to receive intravenous tisagenlecleucel. Eligible patients had progressed after 2 or more lines of prior chemotherapy, and did not respond to or were ineligible for stem cell transplant. 

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Among the 68 patients evaluated for efficacy, the overall response rate (ORR) was 50% (95% CI, 38%-62%), with 32% and 18% of patients reaching complete response (CR) and partial response (PR), respectively. A median duration of response was not yet reached among these patients.

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Grade 3 to 4 cytokine release syndrome — an adverse event (AE) of particular concern in CAR-T cell therapy — was observed in 23% of patients. Other grade 3/4 AEs included neurologic events, encephalopathy, thrombocytopenia, neutropenia, and infection. Other commonly observed AEs included pyrexia, diarrhea, nausea, fatigue, hypotension, edema, and headache.


  1. Kymriah (tisagenlecleucel), first-in-class CAR-T therapy from Novartis, receives second FDA approval to treat appropriate R/R patients with large B-cell lymphoma [news release]. Basel, Switzerland: Novartis; May 1, 2018. Accessed May 2, 2018.