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.
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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.
