Biogen has received authorization from the European Commission (EC) for SKYCLARYS® (omaveloxolone) to treat Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older, marking the first approved treatment for this rare, genetic, neurodegenerative disorder within the European Union. Friedreich’s ataxia, the most common inherited ataxia, manifests early in childhood with symptoms like coordination loss, muscle weakness, and fatigue, progressing to vision and hearing impairments, speech difficulties, and serious complications like diabetes and heart conditions. The disease often leads to wheelchair dependence within 10-20 years, with an average life expectancy of 37 years.
SKYCLARYS, an oral, once-daily medication, demonstrated significant improvements in modified Friedreich Ataxia Rating Scale (mFARS) scores compared to a placebo in the 48-week MOXIe Part 2 trial. Components of the mFARS assessment, including swallowing ability, upper and lower limb coordination, and upright stability, favored SKYCLARYS. Notably, a post hoc analysis showed lower mFARS scores at 3 years for SKYCLARYS-treated patients compared to a natural history group.
Biogen aims to address the unmet needs of the FA community by adding SKYCLARYS to its portfolio, emphasizing its commitment to engage with medical communities and authorities for urgent patient access. Early access programs are open in Germany and France, with plans to expand to additional countries. SKYCLARYS is already approved in the United States, and discussions with regulatory authorities in other regions are underway.
The approval by the EC has been hailed as a significant milestone by various stakeholders, including the Friedreich’s Ataxia Research Alliance (FARA) and Euro-ataxia. Biogen acknowledges the collaborative efforts that contributed to SKYCLARYS development and expresses gratitude to the FA community.
Biogen was founded in 1978 and focuses on innovative biotechnology to transform patient lives. The company takes calculated risks to deliver first-in-class treatments and therapies. The SKYCLARYS approval underscores Biogen’s dedication to advancing breakthrough treatments for rare diseases. Investors are cautioned about potential risks associated with drug development and commercialization, and Biogen commits to updating stakeholders on progress and developments.