Although the neurologic phenotype of FRDA is well-defined, there are currently no established pharmacological treatments. Omaveloxolone, a nuclear factor erythroid 2-related factor 2 (Nrf2) activator, is currently under review by the Food and Drug Administration (FDA) and has the potential to be the first approved treatment for FRDA. In the present report, we have reviewed the basic and clinical literature on Nrf2 deficiency in FRDA, and evidence for the benefit of omaveloxolone.
Sunday, February 5, 2023
Omaveloxolone: An activator of Nrf2 for the treatment of Friedreich's ataxia
Profeta, V., McIntyre, K., Wells, M., Park, C., & Lynch, D. R. (2023). Expert opinion on investigational drugs, 10.1080/13543784.2023.2173063. Advance online publication. doi:10.1080/13543784.2023.2173063
