vatiquinone

Vatiquinone is a first-in-class, orally bioavailable small molecule and selective inhibitor of 15-lipoxygenase (15-LO), developed primarily for the treatment of Friedreich’s ataxia and other inherited mitochondrial diseases. It is derived from vitamin E (alpha-tocotrienol quinone) and acts by targeting 15-lipoxygenase, a key enzyme involved in inflammation, oxidative stress, and ferroptosis—a form of cell death implicated in central nervous system diseases. Vatiquinone also modulates NADPH quinone oxidoreductase 1 (NQO1), enhancing glutathione synthesis to optimize cellular management of oxidative stress and mitochondrial function. The drug has been granted orphan drug designation for several rare diseases including Friedreich’s ataxia, Leigh syndrome, epilepsy, and others. Its development includes data from placebo-controlled trials demonstrating significant slowing of disease progression in Friedreich’s ataxia[1][2][5][6][8].

Brand names

Vincerenone

Other names

vatiquinoneVincerenone