Deus Ex Omaveloxolone: Finding Hope With an Untreatable Illness

I really enjoy the times when I can almost forget about the constant degeneration of my body due to Friedreich’s ataxia (FA). But every so often, I fall out of my wheelchair, notice that my speech is especially muddled, or face another stark reminder I can’t ignore: that my FA symptoms are worsening.

This reality hit me like a slap across the face earlier this week as I was reading my Facebook news feed, of all places. I noticed a question in an online support group for those living with Friedreich’s ataxia: “Any word on omaveloxolone?”

Omaveloxolone (RTA 408), or omav, has become something of a buzzword for those affected by Friedreich’s ataxia. A Phase 2 clinical trial of the investigational oral therapy initially yielded positive results, as Friedreich’s Ataxia News‘ Ana Pena reported in late 2019. This seemed to be a breakthrough in FA research, as no treatment had ever been shown to successfully curb progression of the disease before.

Understandably, omaveloxolone was on the minds of everyone in the FA community after that seemingly positive news was reported. Then, throughout 2020, we waited for the U.S. Food and Drug Administration (FDA) to approve it. 

Sadly, the FDA stated last November that the Phase 2 trial results were not enough to support omaveloxolone’s approval.

Reata, the pharmaceutical company that sponsored the trial, conducted additional studies and shared the results in a press release last March. The company has requested a Type C meeting, a type of meeting in which a sponsor meets with the FDA regarding the development and review of a product, to discuss the study results.

Outside of the FDA’s rulings on treatment approval, FA patients know that any progress would be a game changer for an untreatable, debilitating disease.

Our hearts were broken when the FDA didn’t approve omav, and our hope that it would be the first available FA treatment seemed to be at an end. FA’s progression was here to stay, for now. 

Unexpectedly, hope in omaveloxolone resurfaced last February. The Friedreich’s Ataxia Research Alliance circulated a petition asking Reata to submit a new drug application to the FDA on an urgent basis, and urging the regulatory agency to consider omav’s approval based on existing evidence, as Friedreich’s Ataxia News’ Forest Ray reported at the time.

At the time, more than 74,000 people had signed the petition. The FDA acknowledged receipt of the petition and stated that it remains committed to working with members of the FA community.

For now, we must wait for the results of the Type C meeting. There is no word yet about whether the petition has changed the conversation at the FDA.

I had lost hope in omaveloxolone’s potential availability and hadn’t thought much about it lately until I saw the request for updates on Facebook. 

Another member of the support group answered, “Of course not. They always say a cure [for FA] is coming in the next 5 years. … I’ve lived with FA for over 40 years, and I am no closer to a treatment.”

It may sound harsh, but I fear that commenter may be right. Although current FA research looks very promising, it hasn’t yet translated to anything available that can keep FA from progressing or killing us.

FA patients are waiting on a treatment or cure that may not come in our lifetimes.

Maybe we are jaded, or so accustomed to the idea that treatment will never come, that we prefer pessimism and ennui. Despite that, I don’t think we are hopeless people. The truth is that a treatment’s approval should not be our only hope.

Desperate hope for a treatment or cure to fix everything is seductive and tempting but unrealistic — our deus ex machina. 

Rather than passively waiting on something external to save us, our survival depends on what we do while we wait for one.

The best way to live with FA is to face each moment as it happens. Though we do not have any say about a treatment’s possible approval, we can focus on our own well-being. Are we eating well? Staying active? Going to a doctor regularly? Participating in current clinical trials for FA? Getting enough sleep? How about being a good friend? And letting others know you love them?

Easier said than done, I know. It’s tempting to fantasize about when FA will no longer ravage our bodies. 

Whatever the future holds, having a good life is not about forgetting the bad times, but focusing on happiness in the current moment. A treatment’s approval or denial should not affect that. 

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Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.The opinions expressed in this column are not those of Friedreich’s Ataxia News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.