Friedreich’s ataxia (FA): treatment
Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .
Friedreich’s ataxia (FA): treatment. It is an orally active, small molecule semi-synthetic triterpenoid drug. Fully referenced neurology checklist.
Omeveloxolone
- It is an orally active, small molecule semi-synthetic triterpenoid drug
- It increases antioxidant activity
- It activates the nuclear factor erythroid 2-related factor 2 (Nrf2) pathway
- It is potentially a disease modifying treatment
Treatment of spasticity
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Treatment of neuropathic pain
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Treatment of square wave jerks and ocular flutter
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Treatment of cardiomyopathy
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Management in pregnancy
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Multidisciplinary care
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Investigational treatments
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References
- Corben LA, Lynch D, Pandolfo M, et al. Consensus clinical management guidelines for Friedreich ataxia. Orphanet J Rare Dis 2014; 9:184.
- Lee A. Omaveloxolone: first approval. Drugs 2023; 83:725-729.
- Lynch DR, Farmer J, Hauser L, et al. Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Ann Clin Transl Neurol 2018; 6:15-26.
- Profeta V, McIntyre K, Wells M, Park C, Lynch DR. Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia. Expert Opin Investig Drugs 2023; 32:5-16.
- McCormick A, Shinnick J, Schadt K, et al. Cardiac transplantation in Friedreich ataxia: extended follow-up. J Neurol Sci 2017; 375:471-473.
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