Vatiquinone is the international non-proprietary name for Edison Pharmaceuticals’ EPI-743, an orally bioavailable small molecule being developed by the company for inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. The mechanism of action of EPI-743 involves augmenting the synthesis of glutathione, optimizing metabolic control, enhancing the expression of genetic elements critical for cellular management of oxidative stress, and acting at the mitochondria to regulate electron transport. EPI-743 is a compound being developed by BioElectron (previously known as Edison Pharmaceuticals) to treat Friedreich’s ataxia (FA), a rare, autosomal recessive genetic disorder. The regulation of oxidative stress is disturbed in people with FA. EPI-743 targets NADPH quinone oxidoreductase 1 (NQO1), helping to increase the biosynthesis of glutathione, a compound essential for the control of oxidative stress. The drug does not target any FA-specific biochemical pathways directly, but helps to improve the regulation of cellular energy metabolism in general. Vatiquinone has been investigated for the treatment and prevention of retinopathy, rett syndrome, parkinson's disease, noise-induced hearing loss, and methylmalonic aciduria and homocystinuria, Cblc type. The FDA previously granted orphan drug designation for Edison’s EPI-743 to treat inherited respiratory chain diseases of the mitochondria and Friedreich’s ataxia. The company received orphan drug designation for EPI-743 from the Japanese Ministry of Health, Labour and Welfare and European Medicines Agency Committee on Orphan Products for the treatment of Leigh syndrome.