The Friedreich’s ataxia (FA) community has been abuzz with excitement recently. The first two phases of a clinical trial sponsored by Reata Pharmaceuticals showed that the oral therapy omaveloxolone (RTA 408) is safe and led to improvements in some participating patients.
However, the U.S. Food and Drug Administration (FDA) determined the results of the trial were not enough for approval of the investigational therapy.
Now, members of the FA community are urging Reata to submit a new drug application (NDA) for omaveloxolone to the FDA on an urgent basis, and for the FDA to consider its approval based on evidence from existing clinical trials, BioNews’ Forest Ray reported.
FARA invited “the entire FA community (individuals with FA, family, friends, caregivers, supporters, other rare disease advocates) to sign-on in support.” Supporters can sign the petition until Jan. 20.
FARA’s letter notes that omaveloxolone “has the potential to prevent long-term consequences, slow progression of the disease and improve FA symptoms by addressing the underlying pathologic processes associated with inflammation, mitochondrial dysfunction, and oxidative stress.”
Because no treatments currently are approved for this cruel and progressive disease, our community is desperate for hope and help. The advancement of this investigational therapy provides hope, and it may one day provide help.
As you might recall from previous columns, I have been participating in a clinical trial with omaveloxolone since September 2018. For the first 48 weeks, the trial was double-blind and placebo-controlled. I took three pills every morning, unaware if they were the investigational treatment or a placebo.
After the 48-week trial ended, I was given the opportunity to sign up for the trial’s open-label extension phase. This meant I could take the real investigational treatment rather than a placebo, either until the trial ended or the drug was approved and hit the market, at which point I would pay for prescription medication.
I enrolled in the open-label extension phase in September 2019, and have been taking omaveloxolone at 7 a.m. every day for about 68 weeks. I feel so incredibly blessed and lucky to have this opportunity.
I am in awe of what our relatively small rare disease community has been able to accomplish. With the help of fundraising, over a dozen investigational therapies are currently in the treatment pipeline, and hopefully some of them will one day become available to the FA community.
I am humbly grateful for the scientists and pharmaceutical companies that are partnering with us in the hopes that we can treat and one day cure FA. I am thankful for their dedicated and deliberate work, not only to improve our quality of life but also to save our lives.
I am encouraged by the FA community’s response to the current petition. According to FARA, the letter has received more than 35,000 signatures as of this writing. I hope the letter and the thousands of signatures encourage the powers that be to take the necessary action to approve omaveloxolone for the FA community at large.
As someone who has been on this therapy for over a year, I have high hopes for what it could accomplish. While some of my symptoms have regrettably progressed, the progression of a few symptoms has been paused. Two of my symptoms have even slightly improved since I started taking it.
I know this investigational therapy isn’t a cure for FA. It’s not intended to be. We still have a ways to go before we accomplish that. However, omaveloxolone could become the first ingredient in our cocktail cure for this cruel disease if all goes well.
I hope this also will help to build momentum among other companies and scientists to continue pushing forward with new research.
Regardless of the “what ifs” and my fears about the future, our community needs your help. If you haven’t already, please take a few minutes to sign the letter in support of this potential treatment. It’s a great step forward on our quest to cure Friedreich’s ataxia!
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.
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