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

Skyclarys, OK'd by the FDA today, is being celebrated as a groundbreaking step

Matthew Lafleur avatar

by Matthew Lafleur |

Share this article:

Share article via email
main graphic for column titled

As of today, the status of Friedreich’s ataxia (FA) has changed from untreatable to treatable, thanks to the approval of Skyclarys (omaveloxolone) by the U.S. Food and Drug Administration (FDA).

I still find it unbelievable that FA has a treatment. I’m reminded of high school, when one of my classmates showed up with his foot in a cast, hobbling on crutches. I was strangely jealous that this crutch-dependent guy would eventually get better. Even though most high school bodies tend to repair themselves, maybe with the help of a cast or some crutches, there was nothing I could do to prevent my getting worse. After all, FA, then, was untreatable.

But today, some 20 years later, there is an approved treatment. My teenage pessimism is slowly being replaced by optimism.

Giants in ataxia advocacy share this optimistic mindset.

“This approval results, in part, from decades of the FA community’s efforts,” Kyle Bryant, an athlete and spokesman for the Friedreich’s Ataxia Research Alliance (FARA), told me via text message. “Each dollar raised, clinic visited, letter written to Congress, encouragement voiced to a fellow community member, each feeling of uncertainty that was followed by action. … Every little victory adds up to where we are today. While it is not the finish line, it is a milestone for which we should all feel proud.”

Recommended Reading
Banner for

Living With FA Reinforces My Commitment to Making Progress

Further, FARA co-founder and board president Ron Bartek also celebrated the historic event in an email to me:

“This first FDA approval for FA is a real tribute to and celebration of the FA community — like the patients and families who participated in the clinical trials and natural history study, the FA scientists whose research was essential to this outcome, and our fundraisers and donors that enable us to support that research. We are also grateful to the FDA for listening to our patients’ voices and to Reata [Pharmaceuticals] for being such a steadfast, committed partner. This first treatment is a very important step, and we will continue to work together to further treat and cure FA.”

Finally, I received this message via email: “This is indeed an exciting day for the entire ataxia community, as omaveloxolone represents one of the first FDA approved treatments for any type of ataxia,” said Andrew Rosen, executive director of the National Ataxia Foundation (NAF). “We will continue to work closely with our pharmaceutical partners to accelerate the development of treatments for additional ataxia types, and we thank all of our members who participated in the research and clinical trials for omav.”

The road to a historic therapy

I’m no expert on this new therapy, and the patient and caregiver columns on Friedreich’s Ataxia News aren’t geared toward scientific information. We have an entire news department of journalists with master’s degrees and PhDs who provide that sort of coverage in ways that are easy to understand. Even so, I think it’s prudent to give a basic overview of the drug as I, a layperson with FA, understand it.

Skyclarys, the Reata therapy approved Tuesday, is designed to improve the function of mitochondrial cells that FA weakens. Clinical trials have shown that it significantly slows the progression of the disease.

While taking this medicine may not bring about improvements in an FA patient’s functionality, slowing disease progression is a huge victory. FARA, the stalwart ally of those affected by FA, created a helpful video summary of omaveloxolone’s journey toward FDA approval.

On behalf of the FA community, I’d like to thank Reata for putting time, care, and resources into developing this treatment. It reminds me that not all pharmaceutical companies are only interested in how much money they can make. At this point, I should note that Reata organized the production of a video of me at the Grand Canyon, but I think that helps demonstrate its dedication to the ataxia community.

But even before that, Reata has been a staunch supporter of the ataxia community for years, building a team and participating in many rideATAXIA fundraisers, sponsoring and participating in many yearly FA conferences, and even becoming the lead sponsor of the NAF conference later this year in Las Vegas. It’s worth noting that Reata took that role before Skyclarys was approved.

So what’s next for the FA community?

Regardless of today’s approval, however, there’s still much work to be done within the FA community.

Skyclarys isn’t a cure for FA, but it’s a big next step in the process toward finding one. Therefore, continuing to raise FA research funds and participating in clinical trials of new therapies are essential.

We should also consider that two important groups of FA patients won’t have access to Skyclarys. One of those consists of FA patients younger than 16. Since this drug has only been tested in individuals 16 years of age or older, pediatric studies are needed before Skyclarys can be made available to FA patients of all ages.

The other group includes FA patients living outside of the U.S. Many health organizations in other countries tend to follow the FDA’s approvals, but Reata must still advocate for Skyclarys internationally.

To get this new therapy, FA patients in the U.S. ages 16 or older should talk with their doctor and review Reata’s prescribing information for Skyclarys. The pharmaceutical company has also launched the Reata Education, Access, and Care Helpline, or REACH, to help eligible patients access the therapy.

May the FDA’s approval of Skyclarys be the first domino to fall in the journey toward an eventual cure for FA. As I’ve written before, in FA and in life, always bet on the dark horses.

Little victories matter. And sometimes, we even get to celebrate big ones, too.

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.

A Conversation With Rare Disease Advocates