Biogen’s SKYCLARYS becomes first approved treatment for Friedreich’s Ataxia in EU

In a landmark decision, the European Commission (EC) has authorized Biogen Inc.’s SKYCLARYS (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. This approval heralds SKYCLARYS as the first medication sanctioned within the European Union for this rare, genetic, and progressively debilitating neurodegenerative disease, promising a new dawn in the management and care of FA patients.

Friedreich’s ataxia, the most common form of inherited ataxia, manifests early, typically in childhood, with symptoms that progressively worsen, including coordination loss, muscle weakness, and fatigue. As the disease advances, patients may also face severe complications like vision and hearing loss, speech and swallowing difficulties, diabetes, scoliosis, and critical heart conditions, often leading to wheelchair dependence within 10-20 years of diagnosis and an average life expectancy of 37 years.

Biogen’s development of SKYCLARYS, an oral, once-daily medication, represents a significant breakthrough, having received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, in addition to its Orphan Drug designation in Europe. The EC’s approval is based on compelling efficacy and safety data from the placebo-controlled MOXIe Part 2 trial, where SKYCLARYS demonstrated significant improvements in patients’ modified Friedreich Ataxia Rating Scale (mFARS) scores over placebo.

Sylvia Boesch, the Principal Investigator of the MOXIe study, emphasized the clinical importance of this approval, noting the “clinically meaningful improvements” for patients’ daily lives observed during the trial. Similarly, Priya Singhal, Head of Development at Biogen, highlighted the company’s commitment to bringing this first treatment to people living with Friedreich’s ataxia in Europe, acknowledging the crucial contributions of the FA community to the development and approval of SKYCLARYS.

The Friedreich’s Ataxia Research Alliance (FARA) and Euro-ataxia have welcomed this approval, recognizing it as a pivotal step towards expanding global access to treatment for FA. The collaboration and dedication of researchers, clinical sites, patients, families, and patient organizations have been instrumental in reaching this milestone.

This EC approval not only offers new therapeutic avenues for those affected by FA but also symbolizes hope for a community that has long awaited effective treatment options. As Biogen and the medical community move forward, the focus remains on securing urgent access for patients, underscoring a continued commitment to improving the lives of those living with Friedreich’s ataxia.