European Commission grants approval for first therapy to treat Friedreich’s ataxia

Omaveloxolone will be the first therapy within the European Union to treat Friedreich’s ataxia (FA) in adults and adolescents aged 16 and over.

The company announced yesterday (February 12) that the European Commission has given its approval for the small molecule drug, known as Skyclarys.

Sylvia Boesch is the principal investigator of the MOXIe study and head of the Center for Rare Movement Disorders Innsbruck, Department of Neurology, Medical University Innsbruck, Austria.

She said: “In my clinical practice, I have seen the devastating impact that Friedreich’s ataxia has on patients and their families.

“Friedreich’s ataxia patients treated with Skyclarys in the clinical trial experienced important and clinically meaningful improvements in their daily lives. With this approval, there is optimism within the community that this has the potential to usher in a new era in the management of Friedreich’s ataxia.”

Biogen says Friedreich’s ataxia 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.

“Biogen is proud to add Skyclarys to our portfolio of medicines and address a significant unmet need by bringing the first treatment to people living with Friedreich’s ataxia in Europe,” said Priya Singhal, head of development at Biogen.

“Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients. We sincerely thank the Friedreich’s ataxia community for their contributions that enabled the development of SKYCLARYS and made today’s approval possible.”

The Commission’s approval of Skyclarys 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 the drug 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 Skyclarys over placebo.

Additional exploratory data was provided from a post hoc, propensity-matched analysis in which patients treated with Skyclarys 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.

Jennifer Farmer, Chief Executive Officer of the Friedreich’s Ataxia Research Alliance (FARA), said: “The European Commission approval of Skyclarys is a significant milestone toward expanding global access, bringing the first approved treatment to the Friedreich’s ataxia community in the EU.

“FARA is grateful to all the researchers, clinical sites, individuals with Friedreich’s ataxia and their families, patient organizations, Biogen and the European Medicines Agency for the research, drug development and advocacy efforts that led to this approval. We look forward to continuing our collaboration with the Friedreich’s ataxia community with the goal of expanding access where it is needed.”