Skyclarys TOS

Omaveloxolone (Skyclarys) is a small molecule activator of the Nrf2 (Nuclear factor erythroid 2-related factor 2) pathway, which is the primary cellular defense against oxidative stress. In Friedreich's ataxia, a deficiency in frataxin leads to mitochondrial dysfunction and impaired Nrf2 signaling. By activating Nrf2, omaveloxolone promotes the expression of antioxidant genes, restores mitochondrial membrane potential, and reduces lipid peroxidation. Skyclarys was originally developed by Reata Pharmaceuticals (acquired by Biogen) and is the first FDA-approved treatment for Friedreich's ataxia. The "TOS" (Tablets for Oral Suspension) formulation is a new dosage form designed to improve ease of administration for patients with dysphagia, a common symptom in advanced Friedreich's ataxia.

Brand names

Skyclarys

Other names

omaveloxoloneomaveloxolone tablets for oral suspension