Wednesday, May 28, 2025

Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatment

Gunther, K., Profeta, V., Keita, M. et al. Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatment. Neurol Ther 14, 1105–1114 (2025). doi:10.1007/s40120-025-00749-3

 Most patients with FRDA eventually had access to omaveloxolone, and it was generally well tolerated. Side effects were modest, and, overall, most patients remained on the drug. Abnormalities in serum liver function tests were limited to transaminases, resolved with dose pausing or reduction, and diminished markedly over time. Thus, the safety features of omaveloxolone after administration largely resemble the favorable features noted during clinical trials.