Tuesday, December 10, 2024

Omaveloxolone for the Treatment of Friedreich Ataxia: Efficacy, Safety, and Future Perspectives

Naghipour S, Corben LA, Hulme AJ, Dottori M, Delatycki MB, Lees JG, Lim SY. Omaveloxolone for the Treatment of Friedreich Ataxia: Efficacy, Safety, and Future Perspectives. Mov Disord. 2024 Nov 19. doi: 10.1002/mds.30070. Epub ahead of print. PMID: 39559924. 

The scarcity literature addressing the cardiovascular effects omaveloxolone in FRDA is concerning, especially considering that cardiomyopathy is the primary cause of mortality in affected individuals. Furthermore, the reported potential of omaveloxolone to induce mil elevations in B-natriuretic peptide (BNP), a known marker of heart failure, adds to this concern. Thus, those individuals with FRDA who have cardiomyopathy are advised to monitor BNP levels while using omaveloxolone. In this viewpoint, we examine the current literature on the efficacy and safety of omaveloxolone in treating FRDA, with a focus on its potential impact on cardiovascular health.