Omaveloxolone

Biogen’s therapy works by activating the nuclear factor erythroid 2-related factor 2 pathway. Credit: JHVEPhoto / Shutterstock. Biogen Netherlands has received marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its Skyclarys (omaveloxolone) to treat Friedreich’s ataxia in individuals aged 16 years and above. This approval was granted through a national procedure. Friedreich’s ataxia is a widespread hereditary ataxia type, affecting a minimum of one in every 50,000 individuals. It is characterised by progressive issues with balance and movement. A clinical trial that involved 103 subjects demonstrated that individuals with Friedreich’s ataxia, aged between 16 and 40 years, showed neurological improvement when treated with the therapy versus a placebo. After 48 weeks, those on omaveloxolone experienced a reduction in physical impairment. The oral capsule works by activating the nuclear factor erythroid 2-related factor 2 (Nrf2) pathway. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence This protein is typically found at decreased levels in those with Friedreich’s ataxia and is crucial in aiding cellular response to oxidative stress. UK MHRA Healthcare Quality and Access interim executive director Julian Beach said: “Patient safety is our top priority. I am pleased to confirm the first UK approval for the treatment of Friedreich’s ataxia, omaveloxolone. “We’re assured that the appropriate regulatory standards of safety, quality, and effectiveness for the approval of this new treatment have been met. As with all products, we will keep its safety under close review.” Skyclarys has been indicated for treating this condition in the adult and adolescent population aged 16 years and above, not only in the UK but also in the European Union, Canada, and the US. The US Food and Drug Administration (FDA) has granted the fast track, orphan drug, and rare paediatric disease designations to the therapy, which also gained orphan drug designation from the European Commission for treating Friedreich’s ataxia. In Canada, Skyclarys was authorised for marketing under the Health Canada Priority Review process.

Biogen’s therapy previously secured an orphan drug designation for FA treatment. Credit: Piotr Swat/Shutterstock. Health Canada has granted approval for Biogen’s Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA), a rare, progressive neurodegenerative disease, in individuals aged 16 years and above. The approval, achieved through the priority review process, marks the therapy as the sole treatment in the country targeting the underlying mechanisms of FA. The Canadian regulator’s approval is supported by the pivotal MOXIe Part 2 trial. This placebo-controlled trial included more than 100 subjects. Post 48 weeks, subjects who were given the therapy showed an improvement in their modified FA Rating Scale (mFARS) scores. The oral medication has secured rare paediatric disease, orphan drug and fast track designations from the US Food and Drug Administration (FDA). The European Commission has granted the therapy orphan drug status for the treatment of FA in Europe. FA affects around one in 40,000 people in the country, causing progressive damage to the peripheral nerves and spinal cord, and to the regions of the brain responsible for balance, speech and movement. Symptoms usually appear in childhood or adolescence and progressively worsen. Biogen Canada general manager Eric Tse stated: “The approval of Skyclarys in Canada represents a pivotal moment in our ongoing commitment to advancing treatment options for rare diseases, particularly those with limited treatment options and high unmet needs. “We are incredibly proud to introduce the only Health Canada-approved treatment for FA. We remain steadfast in our dedication to working alongside the medical and patient communities and with payors across Canada to ensure Skyclarys is accessible to individuals living with this challenging condition.” In February 2025, Biogen entered into a collaboration with Stoke Therapeutics, aiming to develop and commercialise zorevunersen for the treatment of Dravet syndrome in all territories outside of Canada, Mexico and the US.