Omaveloxolone treatment slows the progression of Friedreich ataxia (FA), according to a new study published in the Annals of Clinical and Translational Neurology.

The study team reached this conclusion by comparing data from an open-label extension study of omaveloxolone to data from the Friedreich ataxia Clinical Outcome Measures Study (FACOMS), which investigates the natural history of the disease. Their aim was to investigate whether FACOMS can be useful in analyzing data obtained from clinical trials.

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The team, led by Colin Meyer, MD, from Reata Pharmaceuticals in Dallas, Texas, the developers of omaveloxolone, used logistic regression to estimate propensity scores based on gender, age, and modified Friedreich Ataxia Rating scale (mFARS) and gait scores of the patients.

They found that omaveloxolone treatment resulted in persistent benefits over 3  years compared to a matched cohort from FACOMS. 

More precisely, patients in the extension study showed a smaller deterioration in the mFARS score compared to patients in the natural history study.

In a primary pooled population of 136 patients in each group, The mFARS score of those in the natural history study progressed 6.6 points by year 3, compared to 3 points for those treated with omaveloxolone.

“These results detail how propensity-matched analysis may contribute to [the] understanding of effects of therapeutic agents,”  the researchers noted.

“This demonstrates the direct value of natural history studies in clinical trial evaluations,” they added.

FA is a rare genetic disease caused by a mutation in the frataxin gene, which codes for a protein that is essential for ATP synthesis in the mitochondria. 

Omaveloxolone, marketed under the brand name Skyclarys®, is an activator of the nuclear factor erythroid-derived 2-related factor 2. It is thought that it may promote mitochondrial activity, thereby being a potential therapy for FA.


Lynch DR, Goldsberry A, Rummey C, et al. Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data. Ann Clin Transl Neurol. Published online September 10, 2023. doi:10.1002/acn3.51897