Omaveloxolone has a manageable cardiac safety profile in patients with Friedreich’s ataxia (FA), according to a recently published posthoc analysis of cardiac safety assessments during the MOXIe Part 2 study as presented in the 2023 MDA conference.

Cardiomyopathy is a complication experienced by more than two-thirds of patients with FA and is considered the main cause of death associated with FA. Thus, creating a need for the development of drugs capable of treating FA without producing significant adverse effects.

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Omaveloxone is an orally administered drug capable of activating antioxidative transcription factors and inhibiting proinflammatory transcription factors and is currently the only FDA-approved drug specifically for treating FA.

The MOXIe part 2 trial assesses the safety of omaveloxone in patients with FA and mild to moderate cardiac compromise. The study enrolled over 170 participants and started in January 2015.

The randomized, double-blind, placebo-controlled study evaluated omaveloxolone 150 mg once daily for 48 weeks in patients with FA, including those with mild to moderate cardiomyopathy. Results revealed that cardiac adverse events occurred in 9.8% of patients receiving omaveloxolone compared to 13.5% of those receiving a placebo, with no imbalances in arrhythmia-related adverse events. No clinically significant changes were observed between groups in echocardiogram, electrocardiogram, heart rate, and blood pressure assessments.

Although slight increases in BNP and NT-Pro-BNP were observed only in the omaveloxolone treatment group, these adverse effects occurred without signs or symptoms of fluid retention. Mean cholesterol values were higher in the omaveloxolone group than in the placebo group; however, the values remained within normal limits.

The ongoing open-label extension trial has not shown any new cardiac safety signals, and there were no deaths in the study, with the majority of adverse events being mild to moderate in severity.

“Omav was well tolerated and had a manageable cardiac safety profile in MOXIe part 2,” the authors concluded.


Subramony S, Lynch D, Delatycki M,  et al. Assessment of cardiac safety in patients with Friedreich’s Ataxia in the MOXIe trial of omaveloxolone. Poster presented at: 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX