FDA Expands Tisagenlecleucel Indication To Include Relapsed/Refractory B-cell Lymphomas

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The FDA based its approval on results of the single-arm phase 2 JULIET clinical trial.
The FDA based its approval on results of the single-arm phase 2 JULIET clinical trial.

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 (ClinicalTrials.gov 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. 

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.

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. https://www.novartis.com/news/media-releases/kymriahr-tisagenlecleucel-first-class-car-t-therapy-from-novartis-receives-second-fda-approval-treat-appropriate-rr-patients-large-b-cell-lymphoma. Accessed May 2, 2018.

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