PTC-743 Drug

PTC-743 / Vatiquinone (previously EPI-743), is a follow-on or “sister” compound to EPI-A0001, which aims to improve mitochondrial and cellular function by targeting 15-lipooxygenase the key enzyme regulator of a pathway of inflammation, oxidative stress and cell death (known as ferroptosis), which has been implicated in CNS disease, including models of Friedreich’s ataxia. Vatiquinone has been granted Orphan Drug Designation and Fast Track Designation for FA by the U.S. Food and Drug Administration (FDA).