Friedreich ataxia (FA) is an inherited disorder that gets worse over time and affects movement, coordination, and speech. Omaveloxolone (Skyclarys®) capsules are a medicine that has been approved to treat FA in people aged 16 years and older. The approval is based on findings from the MOXIe clinical trial program. This program had 3 parts (Part 1, Part 2, and the MOXIe Open Label Extension). The MOXIe Part 2 study showed that omaveloxolone slowed the worsening of the disease in people with FA compared with those who received an inactive drug–or placebo–over 48 weeks (or 11 months). The study also showed that omaveloxolone causes side effects that were considered manageable, allowing most patients to continue using it.
Monday, August 18, 2025
Propensity-matched analysis comparing omaveloxolone treatment to Friedreich ataxia natural history data: a plain language summary
Lynch, D. R., Goldsberry, A., Rummey, C., Farmer, J., Boesch, S., Delatycki, M. B., … Meyer, C. (2025). Propensity-matched analysis comparing omaveloxolone treatment to Friedreich ataxia natural history data: a plain language summary. Future Neurology, 20(1). doi:10.1080/14796708.2025.2524313
