Omaveloxolone: a groundbreaking milestone as the first FDA-approved drug for Friedreich ataxia

Pilotto, Federica et al., Trends in Molecular Medicine, Volume 0, Issue 0. doi:10.1016/j.molmed.2023.12.002 

 Omaveloxolone represents the first FDA-approved drug for Friedreich’s ataxia (FA). Omaveloxolone targets nuclear factor erythroid 2-related factor 2 (NRF2), which is a master regulator in the antioxidant pathway. The omaveloxolone clinical trial serves as an example for future design of clinical trials. A resolutive cure for FA would probably be achieved only via combinatorial therapy.