VIDEO: Omaveloxolone improves neurologic function in Friedreich’s ataxia patients

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There are several current treatment options for Friedreich's ataxia, or FA as I'll call it for the rest of this time. The most important is what we had before the approval of any drugs. Careful monitoring of neurologic function, cardiac function, presence of diabetes, monitoring for scoliosis and visual loss so that ongoing maintenance care and preventative care can always be carried out. So, we're sure that people with FA don't get other things which affect their disease, or if we see the early signs of heart failure, we can start to treat them. We sometimes still use antioxidants like coenzyme Q even in addition to other prescription treatments. But about two years ago today, the treatment world of FA changed with the approval of omaveloxolone, known commercially as Skyclarys. This is a small molecule which is relatively well tolerated, which is available and approved for people 16 and older, regardless of any other feature of the disease.

It is what I would call a good drug in some people, a very good drug, but not a cure. On average, people will take it, and they'll notice improvement in neurologic function and what actually improves depends on where they are in the disease as their improvement is such it's as if went back a year or a year and a half in time, and then they will stay at that level for a couple years probably before beginning to progress again at a slower rate.

At present, it's not approved for children under 16. It's an unusual drug in that the dose is 150 milligrams. If you cut that dose in half, you lose half the benefit. If you double that dose, you're believed to lose the entire benefit. So it has a very narrow window of dose which is effective. Thus, we don't really know the dose in children. There's studies ongoing right now to try and figure that out, and there will be clinical trials in children coming up very soon. So that's the only approved therapy right now, and really it should be given to anyone, in my opinion, 16 and older, or at least offered the chance.