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

Montreal,QC ., February 28, 2023 – This week  marks the approval of the first medication to treat Friedreich’s Ataxia (FA), a milestone for the FA community  and Ataxia Canada.

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.

“This news is of great significance for  FA patients and their families ,” said Sébastien Huynh, President of Ataxia Canada “ For 50 years, the flame of hope for a life without ataxia, lit by the founder Claude St-Jean, has been burning among the ataxic community . The patient voice and the efforts of FA families and FARA were instrumental in achieving this first approved treatment for FA. Our generous donors have been supporting early discovery research for 50 years now but science is slow and builds progressively. Many patient families in Canada volunteered countless hours to participate in both the MOXIe clinical in the USA trials, as well as the natural history study lead by Dr. Duquette at the CHUM in Montreal in collaboration with 13 other sites across the USA.  Thank you to the global FA community  that advocated for the New Drug Application submission via a petition over 74,000 signatures strong.”

François-Olivier Théberge-Ataxia Canada’s General Manager, added, “There is for the first time an approved treatment for people with FA who are 16 years or older in the USA.We look forward to collaborate with Reata, Health Canada, and other regulatory agencies to give Canadian’s access to this treatment protocol.We hope that this is the first of several approved therapies,” Mr. Theberge continued. “Ataxia Canada has always known that treating FA would require multiple approaches while we wait for genomic therapies. Ataxia Canada is 50 years of constant scientific progress towards a cure. Because science advances, technologies progress, and life is stronger than anything; we look forward to the future “