Omaveloxolone for the Treatment of Friedreich’s Ataxia

Abstract

Friedrich’s ataxia (FRDA), a neurodevelopmental and progressive neurodegenerative disease, is the most common inherited form of ataxia. Omaveloxolone was approved by the US Food and Drugs Administration in early 2023, making it the first treatment available to patients with FRDA. This approval was made possible by combining a compelling cellular mechanism and strong clinical evidence provided through the MOXIe study, the multipart clinical trial evaluating the efficacy of omaveloxolone in patients with FRDA. This review discusses the underlying cellular pathology and proposed mechanism of omaveloxolone in FRDA. The MOXIe study is presented in detail, including a discussion of the challenges faced in clinical trials in FRDA, and rare diseases more broadly. Finally, other therapies under investigation are reviewed briefly.