The results of a clinical trial evaluating the safety and efficacy of omaveloxolone in patients with Friedreich’s ataxia (FA) have shown an acceptable hepatic safety profile, as recently presented at the 2023 MDA conference.
MOXIe part 2 is a double-blind, placebo-controlled trial evaluating omaveloxone in patients with FA. After the trial, patients were free to participate in an open-label extension. In the study, adverse events and hepatic safety parameters were monitored.
Laboratory parameters associated with hepatic function were analyzed, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), and bilirubin levels. The majority of adverse events were mild to moderate in severity. There were no reported deaths in the study.
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The distribution of hepatobiliary disorder adverse events was similar between treatment groups. There were 2 events in the omaveloxolone group and 1 in the placebo group.
ALT increases were reported as adverse events in over 35% of patients in the omaveloxolone group versus approximately 2% of patients in the placebo group.
AST increases were reported as adverse events in over 2% of patients in the omaveloxolone group versus 2% of patients in the placebo group. GGT increases were reported in approximately 6% of patients in the omaveloxolone group compared to no patients in the placebo group.
Discontinuations due to ALT or AST elevation occurred in 1 patient in the omaveloxolone group and no patients in the placebo group.
Most omav-treated patients had maximum ALT and AST increases of more than 3 fold the upper limit. ALT and AST elevations were mild and reversible after drug discontinuation. Maximal values occurred within the first 12 weeks of treatment.
No aminotransferase increase was associated with concurrent increases in total bilirubin, and no Hy’s Law cases were observed.
“Omav was well tolerated and had a manageable hepatic safety profile in clinical studies in patients with FA,” the authors concluded.
Reference
S.H. Subramony, David Lynch,, Martin Delatycki, et al. Assessment of hepatic 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.
