Brand Name: SKYCLARYS™

Chemical Name: Omaveloxolone

Generic Available: No

In 2023, Skyclarys received FDA approval for the treatment of Friedreich’s Ataxia in patients aged 16 and older, making it the first approved treatment for this progressive, life-shortening disease. Friedreich’s Ataxia is caused by a GAA repeat expansion in the FXN gene that reduces production of frataxin, a mitochondrial protein essential for iron regulation and energy production. The resulting frataxin deficiency leads to mitochondrial dysfunction, oxidative damage, and the progressive neurological and cardiac deterioration that defines the disease. Skyclarys is thought to work by activating Nrf2, a key regulator of the body’s antioxidant response, helping to restore mitochondrial function and protect cells from ongoing damage.

Learn more about Skyclarys on Friedreich’s Ataxia Research Alliance’s therapy page.