The application is supported by data from a placebo-controlled study and two long-term studies that included pediatric and adult patients.
Results from the three studies indicated significant, durable, and clinically meaningful evidence of slowing disease progression with vatiquinone.
Vatiquinone is known as a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), "an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia," according to PTC.
