First Gene Therapy Drug Under FDA Expert Panel Review

Tisagenlecleucel could be a one-time treatment for children and young adults with advanced leukemia
Tisagenlecleucel could be a one-time treatment for children and young adults with advanced leukemia

HealthDay News — The potential first gene therapy in the United States is being reviewed by a U.S. Food and Drug Administration expert panel.

At the Wednesday meeting, the panel will assess the safety, effectiveness, and production of the one-time treatment for children and young adults with advanced leukemia, the Associated Press reported. 

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The experimental gene therapy drug was developed by the University of Pennsylvania 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.

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