FDA Approves CAR T-cell Therapy for Pediatric Acute Lymphoblastic Leukemia (ALL)

The US Food and Drug Administration (FDA) has approved Kymriah (tisagenlecleucel) for certain children, adolescents, and young adults with a form of acute lymphoblastic leukemia (ALL). It’s the first gene therapy available in the United States.

Kymriah is a type of CAR T-cell therapy. The therapy is designed to help the body’s own immune system fight cancer. Each dose of the drug is customized to the individual patient. It’s made using a patient’s T-cells, a type of white blood cell that helps fight diseases in the body. The T-cells are collected from the patient and sent to a lab where they are changed in a way that programs them to find and kill the leukemia cells. They are then given back to the patient through an infusion.

The drug is approved for young people ages 3 to 25 years who have B-cell precursor ALL that has not gotten better or has come back after standard treatment.

The FDA based its approval on a small clinical trial of 63 young people with B-cell precursor ALL who had run out of all other options for treatment. The overall remission rate within 3 months of treatment was 83%. The FDA’s action follows a vote in favor of approval by a panel of outside experts.

Kymriah can cause severe side effects

Kymriah comes with a boxed warning for a potentially life-threating syndrome called cytokine release syndrome (CRS), which causes high fever, flu-like symptoms, and potential for serious neurological changes. Other severe side effects include infection, low blood pressure, kidney problems, and decreased oxygen.  CRS and other serious side effects usually occur within the first few weeks after the infusion.

The FDA has also expanded the approval of Actemra (tocilizumab) to treat CRS in children 2 years of age or older. In addition, it has put some extra safety requirements in place. Treatment with Kymriah will be available only at specially certified hospitals and clinics, and medical staff will get special training to manage CRS and serious neurological changes. Not all patients treated with Kymriah will develop CRS and need Actemra. It is important for all patients and their caregivers to understand the signs and symptoms of CRS and know what to do if those symptoms happen.

The FDA approved Kymriah using breakthrough therapy and priority review designations, which are ways to speed up the evaluation of drugs to treat serious conditions.

Kymriah is marketed by Novartis Pharmaceuticals Corp. Actemra is marketed by Genentech, Inc.

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