George Wilmot, MD, PhD, describes key findings from Skyclarys (omaveloxolone) trials and how that translates into changes a healthcare provider might see in a Friedreich’s ataxia patient.

Well, there were positive findings for Skyclarys, and that’s an exciting thing. It seemed like there’s some decrease in progression rate by a number of different measures that were used.

The symptomatic benefit is a little harder to say. The measurements of that and the anecdotal experience sometimes doesn’t exactly line up, I think.

My personal feeling is because the benefits on a symptomatic basis — rather than progression of the disease measurement as we measured it in the studies — is harder to nail down, that it’s harder to count on any kind of a symptomatic benefit, although some patients do report that. 

The study itself measured the disease through the scale — the mFARS — and saw that, versus placebo in the initial phase, there were improvements. And then that improvement versus placebo was maintained in the extension phase, when everybody was exposed to Skyclarys.

For that reason, we think that there is a decrease in the progression rate of the disease. Whether or not that’s maintained over a longer period of time is not very well known right now. But at least for the beginning phase — the first couple of years, few years — it looks like there’s a decrease in progression rate, and that is maintained while you’re on the medicine.