FDA Approves First Gene Therapy in the US

The FDA has approved Kymriah (tisagenlecleucel), the first gene therapy available in the United States, for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Kymriah is a genetically-modified autologous T-cell immunotherapy that is approved for patients aged 25 years or younger with B-cell precursor ALL that is refractory or in second or later relapse. Doses of Kymriah 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.
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The safety and efficacy of the treatment option was evaluated in a multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. Overall remission rate within 3 months was 83%.

Potential adverse effects include serious infections, low blood pressure, acute kidney injury, fever, and decreased oxygen. Kymriah also includes a boxed warning for cytokine release syndrome.

Due to these risks, the FDA is requiring that hospitals and clinics that dispense Kymriah be specially certified, and has approved the treatment with a risk evaluation and mitigation strategy. Further evaluation of long-term safety is being evaluated in a post-marketing observational study.

—Michael Potts

Reference:

FDA approval brings first gene therapy to the United States [press release]. August 30, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery.