Biogen's Skyclarys Approved by Health Canada for Friedreich's Ataxia

Health Canada has granted approval for the use of Biogen’s medication, Skyclarys (omaveloxolone), as a treatment for Friedreich’s ataxia (FA), a rare and progressively worsening neurodegenerative disorder. This approval is specifically for individuals aged 16 and older. Importantly, Skyclarys is now recognized as the only available treatment in Canada that targets the underlying causes of FA, thanks to a priority review process.

The foundation for Health Canada’s decision lies in the results of the MOXIe Part 2 trial, a pivotal study involving over 100 participants. This placebo-controlled trial demonstrated that after 48 weeks, those who received the medication showed significant improvements in their modified FA Rating Scale (mFARS) scores. This evidence underscores Skyclarys’ potential in positively impacting the health of individuals with FA.

Skyclarys has also garnered several designations from the US Food and Drug Administration (FDA), including rare pediatric disease, orphan drug, and fast track designations. Additionally, the European Commission has recognized the therapy as an orphan drug for treating FA across Europe, highlighting the global recognition of this treatment's significance.

Friedreich’s ataxia is a condition that affects approximately one in 40,000 individuals in Canada. It is characterized by the gradual deterioration of peripheral nerves, the spinal cord, and specific brain regions that govern balance, speech, and movement. Symptoms commonly emerge in childhood or adolescence and progressively become more severe, presenting significant challenges for those affected.

Eric Tse, the general manager of Biogen Canada, expressed his enthusiasm over the approval of Skyclarys in Canada. He emphasized the significance of this event in Biogen’s ongoing efforts to enhance treatment options for rare diseases, particularly those with scarce treatment alternatives and substantial unmet needs. Tse stated, “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.”

Furthermore, Biogen has been actively engaged in collaborative efforts to extend its therapeutic reach. In February 2025, the company entered into a partnership with Stoke Therapeutics to develop and commercialize zorevunersen for treating Dravet syndrome. This collaboration intends to cover all territories outside Canada, Mexico, and the US, marking a strategic move to broaden Biogen’s therapeutic portfolio and impact.

In summary, the approval of Skyclarys signifies a major advancement in the treatment landscape for Friedreich’s ataxia in Canada. This development not only offers hope to individuals battling this debilitating disease but also reinforces Biogen’s commitment to addressing rare diseases with limited treatment options. The collaboration with Stoke Therapeutics further exemplifies Biogen’s strategy to enhance its offerings and reach in the realm of rare neurological disorders.