CDA Issues Positive Reimbursement Recommendation for SKYCLARYS™ (omaveloxolone), the Only Health Canada-Approved Treatment for Managing Friedreich Ataxia

• Final CDA recommendation reflects the urgent unmet need in a progressive, life-limiting condition and recognizes the value of a therapy that slows disease progression
• Regional differences in disease burden make focus on Quebec and Eastern Canada especially important

TORONTO, July 29, 2025 /CNW/ - Biogen Canada Inc. is pleased to announce a significant step forward for Canadians living with Friedreich ataxia (FA), with the positive reimbursement recommendation by Canada's Drug Agency (CDA) Canadian Drug Expert Committee (CDEC) for SKYCLARYS™ (omaveloxolone) as a treatment for patients 16 years of age and older who meet certain criteria.¹ This milestone brings Canadians outside Quebec affected by this rare neurodegenerative disease a step closer to accessing the only approved therapy for the condition.

The CDA recommendation is based on an assessment of clinical trial and economic evidence with input from physicians, patients and caregivers. The clinical evidence presented was from the Phase II MOXIe Part 2 trial, which showed that SKYCLARYS significantly slowed neurological decline compared to placebo as measured by the modified Friedreich Ataxia Rating Scale (mFARS), a tool used to assess neurological decline and disease progression. The individuals enrolled in the clinical trial were monitored over a 48-week period and showed reduced decline in their abilities when performing activities of daily living, which are essential for maintaining independence.²

"For individuals affected by this progressive genetic disease, even a modest slowing in disease progression can have a profound impact on their lives," said Stacey Lintern, Chief Executive Officer, Muscular Dystrophy Canada. "Friedreich ataxia is a life-limiting condition that has been marked by a lack of treatment options and longstanding unmet needs in Canada. The CDA recommendation represents an important step toward changing that reality by making treatment more accessible and offering patients and families much-needed choice in managing their condition. Advancements like SKYCLARYS bring renewed hope to patients and their families."

A progressive and life-shortening genetic disease, FA has a devastating impact on those diagnosed, reducing quality of life and limiting life expectancy to an average of 37 years.^3,4,5 In Canada, it is estimated to affect 1 in 40,000 individuals.⁶

"We are encouraged by the CDA's recognition of SKYCLARYS as vital therapy for the Friedreich ataxia community, representing critical progress for this life-altering disease in urgent need of treatment options. In regions like Quebec and Eastern Canada, where more people are affected by FA, the need for timely access to new treatment options is especially urgent," said François-Olivier Théberge, General Manager of Ataxia Canada. "FA takes a heavy toll—people and families living with the disease face significant and often overwhelming challenges. For over 53 years, Ataxia Canada has been driven by the vision of our founder, Claude St-Jean, who believed deeply in the power of science and community to bring hope to those affected by rare diseases. We remain committed to his mission to advocate for equitable access in all provinces, including Quebec, and look forward to helping ensure that all Canadians living with Friedreich ataxia have a fair chance to benefit from SKYCLARYS."

The CDA recommendation marks an important turning point in improving treatment options for individuals living with Friedreich ataxia.

"This milestone represents much needed progress in addressing a longstanding unmet need for Canadians living with Friedreich ataxia," said Eric Tse, General Manager, Biogen Canada Inc. "Biogen is proud to be at the forefront of innovation in rare and serious neurodegenerative diseases, and remains committed to working collaboratively with healthcare systems and decision makers across Canada to support timely and equitable access to a treatment that will set the new standard of care in the management of FA."

About Biogen

Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient 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.

Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit www.biogen.ca. 

References:

1. Canada's Drug Agency (CDA). Reimbursement Recommendation (Final) Omaveloxolone (Skyclarys). Available at: https://www.cda-amc.ca/sites/default/files/DRR/2025/SR0864-Skyclarys_Rec.pdf Accessed July 2025.
2. Regulatory Decision Summary – Skyclarys. Regulatory Decision Summary for Skyclarys - Drug and Health Products Portal. Accessed 17 July 2025.
3. Parkinson MH, Boesch S, Nachbauer W, Mariotti C, Giunti P. Clinical features of Friedreich's ataxia: classical and atypical phenotypes. J Neurochem. 2013 Aug;126 Suppl 1:103-17. doi: 10.1111/jnc.12317. PMID: 23859346.
4. Tsou AY, Paulsen EK, Lagedrost SJ, Perlman SL, Mathews KD, Wilmot GR, Ravina B, Koeppen AH, Lynch DR. Mortality in Friedreich ataxia. J Neurol Sci. 2011 Aug 15;307(1-2):46-9. doi: 10.1016/j.jns.2011.05.023. Epub 2011 Jun 8. PMID: 21652007. 
5. Corben LA, Collins V, Milne S, Farmer J, Musheno A, Lynch D, Subramony S, Pandolfo M, Schulz JB, Lin K, Delatycki MB; Clinical Management Guidelines Writing Group. Clinical management guidelines for Friedreich ataxia: best practice in rare diseases. Orphanet J Rare Dis. 2022 Nov 12;17(1):415. doi: 10.1186/s13023-022-02568-3. PMID: 36371255; PMCID: PMC9652828. 
6. SickKids. Friedreich ataxia (FRDA). Available at: https://www.aboutkidshealth.ca/friedreich-ataxia-frda Accessed July 2025.

SOURCE Biogen Canada Inc.

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