Category: Ataxia
Objective: The aim was to monitor safety and efficacy of omaveloxolone treatment in a single center real life cohort, focusing on Friedreich Ataxia (FRDA) patients that were not reflected by the study population.
Background: Friedreich Ataxia is the most frequent autosomal recessive hereditary ataxia. Based on the outcomes of a randomized placebo-controlled phase III trial, omaveloxolone has received approval for treatment from the age of 16 years on in multiple countries worldwide.
Method: FRDA patients were assessed at baseline, 3 and 12 months after initiation of omaveloxolone treatment. Additional safety laboratory assessments were carried out monthly within the first 3 months and after clinical judgment. Clinical course was monitored by the modified Friedreich ataxia rating scale (mFARS). Laboratory assessments included liver enzymes, lipid values and proBNP. Routine cardiac assessment (ECG, Echocardiography) was performed annually. FRDA patients that received omaveloxolone within a clinical study were excluded from the analyses.
Results: 22 FRDA patients with a mean age of 45 years, a disease duration of 24 years and a GAA repeat length of 500 were included in this analyses. mFARS at baseline showed a mean value of 43,72 points, decreased slightly after 3 months and returned near baseline after 12 months of treatment. Monitoring of liver enzyme parameters showed marked ALT and AST increase with a maximum 1 month after treatment initiation without evidence of clinical liver damage. LDL values increased gradually peaking at month 3, returning to baseline after 12 months, while HDL decreased slightly. No cardiac issues were observed during this period. Two patients stopped treatment, one each because of gastrointestinal side effects and muscle cramps.
Conclusion: Omaveloxolone treatment in this single center open label cohort shows stabilization of neurological function after 1 year. The side effect profile is comparable to previously published studies. The less pronounced effect in neurological functions may be attributed to different clinical progression and ceiling effects of rating scales in this cohort, not reflecting the study population of the RCT trial.
To cite this abstract in AMA style:
W. Nachbauer, M. Amprosi, D. Boesch, M. Schranz, E. Indelicato, L. Schwaighofer, S. Boesch. Omaveloxolone in a real-world setting: implications in Friedreich Ataxia beyond the classic study population [abstract]. Mov Disord. 2026; 41 (suppl 1). https://www.mdsabstracts.org/abstract/omaveloxolone-in-a-real-world-setting-implications-in-friedreich-ataxia-beyond-the-classic-study-population/. Accessed October 1, 2026.« Back to 2026 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/omaveloxolone-in-a-real-world-setting-implications-in-friedreich-ataxia-beyond-the-classic-study-population/