HealthDay News — The US Food and Drug Administration is reviewing the potential first gene therapy in the United States.

The panel assessed the safety, effectiveness, and production of the one-time treatment for children and young adults with advanced leukemia, the Associated Press reported.

The experimental gene therapy drug was developed by the University of Pennsylvania (UPenn) and Novartis and is called tisagenlecleucel (CTL019). 

It involves reprogramming a patient’s own T cells with a transgene encoding a chimeric antigen receptor to identify and eliminate CD19-expressing malignant and non-malignant cells.


After reviewing the data, the panel will vote on whether to recommend FDA approval, the AP reported. A final decision from the FDA is expected later this year. 

Reference 

Tisagenlecleucel (CTL019) for the Treatment of Pediatric and Young Adult Patients With Relaped/Refractory B-Cell Acute Lymphoblastic Leukemia [press release]. Washington, DC: US Food and Drug Administration; July 12, 2017.

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