May 01, 2018—Today, the U.S. Food and Drug Administration (FDA) expanded the approval of the chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma following two or more lines of systemic therapy.
This expanded indication follows the initial approval of tisagenlecleucel for acute lymphoblastic leukemia in August of last year. Today’s approval includes diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. This marks the third ever approval/indication of a CAR T-cell therapy.
Today’s approval is based on results from the phase II JULIET study (NCT02445248), recently presented at the 2017 ASH Annual Meeting & Exposition. The CD19-directed therapy demonstrated an overall response rate of 50% (95% CI, 38%-62%) in adult patients with R/R DLBCL, including a complete response rate of 32% and a partial response rate of 18%. The median duration of response had not been reached.
Tisagenlecleucel is a patient-specific, one-time treatment that requires a patient’s lymphocytes to be collected and genetically modified. During the manufacturing process a gene containing the CAR protein is introduced, allowing the direction of T cells to cancerous cells containing the surface antigen CD19.
Treatment with tisagenlecleucel is associated with an increased risk of cytokine release syndrome (CRS). In JULIET, grade 3/4 CRS occurred in 23% of patients. Additionally, 18% of all infused patients experienced grade 3/4 neurologic events, including 11% of patients who experience severe encephalopathy. Grade 3/4 thrombocytopenia lasting more than 28 days occurred in 40% of patients; neutropenia in 25%. Infections occurred in 25% of patients. Other common adverse events (AEs) included pyrexia, diarrhea, nausea, fatigue, hypotension, edema and headache.
Jonathan A. Bell
Published Online: Tuesday, May 01, 2018
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Tisagenlecleucel approved for second indication: Large B-cell lymphoma
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