First gene therapy in the U.S. approved by the FDA

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In Washington, D.C., the U.S. Food and Drug Administration made the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases. The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. In one clinical trial, the remission rate within three months of treatment was 83 percent.