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.

Rare Disease or Not, Only I Can Define My Success

I’m approaching what can easily be labeled the midpoint of life for the so-called average person. Of course, such averages rarely take rare disease into account. But even without the progression of Friedreich’s ataxia (FA), living to (almost) 40 is no easy task. I mean, just think about…

I’m Learning to See Myself as an Ataxia Warrior

I’ve never thought of myself as especially strong, or as someone who could identify with a warrior’s personality. Because I face the progressively debilitating disorder Friedreich’s ataxia, I sadly realized that my physical strength, like the rest of my capabilities, will dwindle over time much more quickly than…