FDA approval of 1st FA treatment is a reminder of my mission

For Sean Baumstark, Skyclarys is a sign that 'we're going to beat this thing'

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by Sean Baumstark |

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It may be old news by now, but for those of us living with Friedreich’s ataxia (FA), there is a particular buzz about the very first treatment for FA approved by the U.S. Food and Drug Administration, Skyclarys, formerly known as omaveloxolone. I don’t believe the excitement surrounding this approval will fade anytime soon.

Developed by Reata Pharmaceuticals, Skyclarys is a once-daily oral treatment indicated for treating FA in adults and adolescents ages 16 and older. The recommended dosage is 150 mg, taken in three 50 mg capsules. Reata expects Skyclarys to be available in the second quarter of this year.

Although I love my life and have experienced many exciting things, this approval, announced on Feb. 28, is the best news I’ve seen following my diagnosis of FA in 2007.

Before that, I didn’t know anything about this disease, and I certainly wasn’t familiar with anyone living with it or otherwise connected to the FA community. This is a common theme among those of us in the rare disease world. As soon as my doctor explained to me what I was up against, as best he knew how, feelings of loneliness, despair, and hopelessness came rushing in.

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A patient’s perspective on the 1st Friedreich’s ataxia treatment

Hope, constantly renewed

Thankfully, those emotions faded quickly as I met other people with FA, as well as professionals who knew about the work being done to discover treatments and eventually a cure. Although FA has changed my life, my hope has been constantly renewed after I joined my first FA fundraiser in Bakersfield, California, just three weeks after my diagnosis.

I am privileged to know dozens of people who are dedicated to participating in research and fundraising. Every time I have the chance to visit with them, they seem to possess an infectious enthusiasm that declares, “We’re going to beat this thing.” People living with FA, as well as their family members, friends, doctors, scientists, and even random neighbors have joined the war against this disease.

With the approval of Skyclarys, the entire FA community has delivered its most significant strike to date in our war.

While a cure and new therapies to reverse the damage caused by FA aren’t yet in sight, we’re closer than ever. Skyclarys serves as a reminder that we’re going to beat this thing.

Unfortunately, there are other dramatic reminders, too, such as a near fall I had yesterday. I realize that we aren’t finished with our work. FA is real and requires my attention, awareness, and respect.

Collectively, though, we’re making progress, and we will win.

Note: 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.


Annelies Swan avatar

Annelies Swan

Thank you for your uplifting article, it cheered me up tremendously

I was diagnosed with FA in 2008 and my daily struggles have increased lately. I live in the UK
and hope that this new FA drug will be approved in the UK very soon

Keep up the good work and thank you so much for your contributions


Sandr Terpstra avatar

Sandr Terpstra

Great article, I got diagnosed in 2003, I live in NZ and can’t wait to get hold of this new drug. Have you noticed any changes or side effects since taking this drug?


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