FDA Approves First Gene Therapy


Yesterday, the U.S. Food and Drug Administration approved the first gene therapy – Kymriah – for young adult and pediatric patients with a specific type of acute lymphoblastic leukemia (ALL). This historic approval paves the way for a new approach to treat other types of cancer and serious diseases.

ALL, the most common childhood cancer in the United States, is a cancer of the bone marrow and blood in which the body forms abnormal lymphocytes (a type of white blood cell). The disease often progresses quickly. Kymriah reprograms a patient’s own immune system cells (called T cells) to attack and destroy abnormal cancer cells.

According to FDA Commissioner Scott Gottlieb, M.D., gene and cell therapies have the potential to transform medicine, significantly improving the ability to treat – and potentially cure – a number of life-threatening conditions. Kymriah is approved for ALL patients 25 years and younger whose disease has not responded to treatment or has relapsed.

Sourced from: FDA