FDA approves first gene therapy

FDA approval brings first gene therapy to the United States for treating cancer and other life-threatening diseases.

The U.S. Food and Drug Administration has approved the first gene therapy treatment in the USA, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The FDA approved Kymriah (tisagenlecleucel) for certain paediatric and young adult patients with a form of acute lymphoblastic leukemia.

New technologies such as gene therapies hold out the potential to treat and even cure many intractable illnesses.

The safety and efficacy of Kymriah were demonstrated in clinical trials. But treatment has the potential to cause severe side effects.

Because of the risk, Kymriah is approved with a risk evaluation and mitigation strategy, which includes elements to assure safe use. The FDA must specially certify hospitals and clinics that dispense Kymriah. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events.