Omaveloxolone: potential new agent for Friedreich ataxia

Neurodegener Dis Manag. 2021 Apr;11(2):91-98. doi: 10.2217/nmt-2020-0057. Epub 2021 Jan 12.


Friedreich ataxia is a slowly progressive neurodegenerative disorder leading to ataxia, dyscoordination, dysarthria and in many individuals vision and hearing loss. It is associated with cardiomyopathy, the leading cause of death in Friedreich ataxia (FRDA), diabetes and scoliosis. There are no approved therapies, but elucidation of the pathophysiology of FRDA suggest that agents that increase the activity of the transcription factor Nrf2 may provide a mechanism for ameliorating disease progression or severity. In this work, we review the evidence for use of omaveloxolone in FRDA from recent clinical trials. Though not at present approved for any indication, the present data suggest that this agent acting though increases in Nrf2 activity may provide a novel therapy for FRDA.

Keywords: Nrf2; antioxidant response element; ataxia; cardiomyopathy; glutathione; reactive oxygen species; scoliosis.

Plain language summary

Lay abstract Friedreich ataxia is a progressive neurological disorder associated with the loss of the ability to walk, loss of hand coordination and loss of speech. Patients also develop heart disease that can be fatal. The disorder results from a relative lack of the protein frataxin, which causes abnormal mitochondrial function and lack energy production and thus gives rise to the features of the disease. Recent evidence suggests that omaveloxolone may reverse some of these features, leading to stabilization of disease progression. This drug represents a potential new therapy for Friedreich ataxia.

MeSH terms

  • Friedreich Ataxia / drug therapy*
  • Humans
  • Triterpenes / therapeutic use*


  • Triterpenes
  • omaveloxolone