RARE Daily

EC Approves Biogen’s Skyclaris, First Therapy in EU to Treat Friedreich’s Ataxia

February 13, 2024

Rare Daily Staff

The European Commission approved Biogen’s Skyclaris for the treatment of the progressive, neurodegenerative condition Friedreich’s ataxia.The approval for Skyclaris is for use in adults and adolescents aged 16 years and older. It is the first treatment approved within the European Union for the condition.

Friedreich’s ataxia (FA) is the most common inherited ataxia. Early symptoms typically appear in childhood and include progressive loss of coordination, muscle weakness, and fatigue. As the disease progresses, people living with FA may also experience vision impairment, hearing loss, problems with speech and swallowing, diabetes, scoliosis, and serious heart conditions. Many people with FA use walking aids, and often require a wheelchair within 10-20 years following their diagnosis. Unfortunately, complications from FA contribute to a life expectancy of 37 years on average.

Skyclaris is an oral, once-daily medication indicated for the treatment of FA in adults and adolescents aged 16 years and older in the U.S. and European Union. Skyclaris received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration. The European Commission granted Orphan Drug designation in Europe to Skyclaris for the treatment of FA.

“In my clinical practice, I have seen the devastating impact that Friedreich’s ataxia has on patients and their families,” said Sylvia Boesch, principal investigator of the MOXIe study and head of the Center for Rare Movement Disorders Innsbruck, Department of Neurology at the Medical University in Innsbruck, Austria. “Friedreich’s ataxia patients treated with Skyclaris in the clinical trial experienced important and clinically meaningful improvements for their daily lives.”

The EC approval of Skyclaris is based on efficacy and safety data from the placebo-controlled MOXIe Part 2 trial. At the end of the 48-week study, patients who received Skyclaris had significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores relative to placebo. All components of the mFARS assessment, including ability to swallow (bulbar), upper limb coordination, lower limb coordination, and upright stability, favored Skyclaris over placebo. Additional exploratory data was provided from a post hoc, propensity-matched analysis in which patients treated with Skyclaris in MOXIe (Extension) had lower mFARS scores at 3 years, as compared to a matched natural history group. The most common side effects are increased liver enzymes, decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.

“The European Commission approval of Skyclaris is a significant milestone toward expanding global access, bringing the first approved treatment to the Friedreich’s ataxia community in the EU,” said Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance.

Stay Connected

Sign up for updates straight to your inbox.