FDA Breakthrough: First Ever CAR-T Therapy Approved

Submitted on 08/30/17

The US Food and Drug Administration (FDA) has made the first gene therapy available, marking their approval of a new approach to the treatment of cancer.

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Tisagenlecleucel (Kymriah, Novartis) is a cell-based gene therapy indicated for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. Doses of tisagenlecleucel are customized using individual’s own T-cells. The T-cells are sent to a manufacturing center where they are genetically modified to include a chimeric antigen receptor (CAR) which causes the T-cells to target leukemia cells with the CD19 antigen.

The approval came following safety and efficacy data from a multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. Overall remission rate among these patients within three months of tisagenlecleucel treatment was 83%.

Treatment with tisagenlecleucel has the potential to cause cytokine release syndrome (CRS), as indicated on its boxed warning. The FDA today also expanded the indication of tocilizumab (Actemra) to include CAR T-cell-induced severe or life-threatening CRS in patients aged 2 years or older.

Other side effects associated with tisagenlecleucel include serious infections, hypotension, acute kidney injury, fever, and hypoxia.—Zachary Bessette