Omaveloxolone for the treatment of Friedreich’s ataxia

Omaveloxolone is in development for the treatment of patients with Friedreich’s Ataxia (FA). FA is an inherited disease that causes a range of symptoms including difficulty walking, inability to coordinate movements, muscle weakness, speech problems, damage to the heart muscle and diabetes. Patients with FA do not have enough frataxin, a protein that regulates iron in mitochondria (the energy-producing components of cells). As a result, iron builds up within the cells, which in turn results in the production of toxic forms of oxygen that damage cells in the brain, the spinal cord, and nerves, as well as in the heart and pancreas.