U.S. Food & Drug Administration Approves Reata Pharmaceuticals’ SKYCLARYS™ (Omaveloxolone) for the Treatment of Adults and Adolescents Aged 16 Years and Older with FA

DOWNINGTOWN, Pa., February 28, 2023 – Today marks the approval of the first medication to treat Friedreich’s Ataxia (FA), a milestone for the FA community and the Friedreich’s Ataxia Research Alliance (FARA). Reata Pharmaceuticals announced that the U.S. Food & Drug Administration has approved SKYCLARYS™ (omaveloxolone) for the treatment of people who have FA and are 16 years of age or older. This announcement comes on Rare Disease Day, which is recognized as a day to raise awareness of the unmet medical needs for the hundreds of millions of people living with rare diseases worldwide.

FA is a rare, progressive neuromuscular disease that affects an estimated 15,000 children and adults globally. It typically begins with difficulties in balance and coordination and rapidly progresses over a short period of time, leading to a life-altering loss of mobility and energy. FA also presents a serious risk of diabetes and life shortening cardiac disease.

The results of the MOXIe Part 1 and 2 clinical trials and the open-label extension study in people confirmed to have FA demonstrate mechanism of action, efficacy, and safety. The key previously reported outcomes include:
  • The MOXIe Part 2 clinical trial data showed that after one year, individuals on treatment with SKYCLARYS compared to placebo had an improvement in neurological symptoms.
  • The longer-term open-label extension study, where individuals with FA were taking SKYCLARYS for 3 years, showed a >50% slowing of progression compared to closely matched individuals in the FA natural history study.
  • Favorable tolerability and safety profile in the MOXIe Part 1 and 2 clinical trials and the open-label extension study in people with FA.
“Today is a true celebration of the power of the patient community,” said Ron Bartek, FARA Co-Founder and President. “The patient voice and the efforts of FA families were instrumental in achieving this first approved treatment for FA. Our generous donors supported early discovery research with funding from the FARA Grant Program. Our patient families volunteered countless hours to participate in both the MOXIe clinical trials, as well as the natural history study, and our global FA community advocated for the New Drug Application submission via a petition over 74,000 signatures strong.”

Mr. Bartek continued, “This is the collaborative effort and incorporation of the patient voice to advance therapeutic development that we envisioned when we founded FARA. We are grateful to Reata and FDA for being attentive to the lived experience of the FA community. SKYCLARYS offers hope for those with FA and their families, and we are thrilled that this treatment will now be available.”

Jen Farmer, FARA’s Chief Executive Officer, added, “Today, there is an approved treatment for people with FA who are 16 years or older. They will no longer be told there is no treatment for the condition. We look forward to continued collaboration with Reata, FDA, and other regulatory agencies to expand access to broaden that group to include all FA patients regardless of age or location.”

“We are hopeful that this is the first of several approved therapies,” Ms. Farmer continued. “FARA has always known that treating FA would require a cocktail of multiple approaches. We hope that the benefits demonstrated in the MOXIe Part 2 trial and open-label extension study give individuals with FA improved quality of life and present a first treatment in the cocktail of therapies to ultimately slow, stop, and reverse FA. We are excited that there is an established path to approval, and we remain committed to bringing effective therapies forward for all people living with FA.”

For Reata Pharmaceuticals’ full press release, click here.
For more information on the REACH program, click here.
For additional information, visit FARA's Research Pipeline.