[1][4] Friedreich's ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, poor balance, difficulty walking, changes in speech and swallowing, and a shortened lifespan.

[9] Nrf2 transcriptionally regulates multiple genes that play both direct and indirect roles in producing antioxidative potential and the production of cellular energy (i.e., adenosine triphosphate or ATP) within the mitochondria.

Preclinical studies have demonstrated that omaveloxolone possesses antioxidative and anti-inflammatory activities[9][11] and the ability to improve mitochondrial bioenergetics.

[10] Omaveloxolone is under clinical investigation for a variety of indications, including Friedreich's ataxia, mitochondrial myopathies, immunooncology, and prevention of corneal endothelial cell loss following cataract surgery.

[4] The primary objective was to evaluate the change in the modified Friedreich's Ataxia Rating Scale (mFARS) score compared to placebo at week 48.