CAMBRIDGE, Mass. — Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.
“In my clinical practice, I have seen the devastating impact that Friedreich’s ataxia has on patients and their families,” said Sylvia Boesch, M.D., MSc, Principal Investigator of the MOXIe study and Head of the Center for Rare Movement Disorders Innsbruck, Department of Neurology, Medical University Innsbruck, Austria. “Friedreich’s ataxia patients treated with SKYCLARYS in the clinical trial experienced important and clinically meaningful improvements for their daily lives. With this approval, there is optimism within the community that SKYCLARYS has the potential to usher in a new era in the management of Friedreich’s ataxia.”
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, M.D., M.P.H., 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 EC 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 SKYCLARYS 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.
“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,” said Jennifer Farmer, Chief Executive Officer of the Friedreich’s Ataxia Research Alliance (FARA). “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.”
“On behalf of Euro-ataxia and its members, I am delighted to welcome the European Commission approval of SKYCLARYS, a much-needed medicine for adults with Friedreich’s ataxia in EU countries,” said Andreas Nadke, President of Euro-ataxia. “Our member’s patient groups have been working and waiting for this day for many years, and we firmly believe that this will be a successful and gratifying beginning in the treatment of Friedreich’s ataxia.”
For detailed product information, please see the Summary of Product Characteristics on the European Medicines Agency website at www.ema.europa.eu. Biogen is committed to working closely with all stakeholders to ensure that eligible European patients can have access to this treatment. Early access programs for SKYCLARYS are currently open in Germany and France, with plans to expand to additional countries where possible. SKYCLARYS is also approved for use in the United States, and Biogen is engaging with regulatory authorities in other regions.
About SKYCLARYS® (omaveloxolone)
SKYCLARYS® (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older in the U.S. and European Union. SKYCLARYS 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 SKYCLARYS for the treatment of FA.
About Friedreich’s Ataxia
Friedreich’s ataxia (FA) is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder. It is the most common inherited ataxia. Early symptoms of FA, such as progressive loss of coordination, muscle weakness and fatigue, typically appear in childhood and can overlap with other diseases. Most people living with FA will need to use a wheelchair within 10-20 years of their first symptoms. The reported average age of death for FA patients is just 37 years old, although with appropriate and targeted care, individuals may live many years after confinement to a wheelchair.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient’s lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.
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