SKYCLARYS™ (omaveloxolone) Approval by Health Canada Ushers in a New Era for Friedreich's Ataxia Treatment in Canada Français

News provided by

Biogen Canada Inc.

Mar 17, 2025, 07:00 ET

  • As the only approved treatment to address disease progression, SKYCLARYS addresses a long-standing gap in the care of Canadians living with Friedreich's ataxia
  • Friedreich's ataxia is a rare, progressive, debilitating and life-shortening neurodegenerative disease1
  • The approval of SKYCLARYS reflects Biogen's commitment to pioneering innovation in rare diseases, driving progress in patient care, and addressing critical unmet needs

TORONTO, March 17, 2025 /CNW/ - Biogen Canada Inc. is pleased to announce that Health Canada has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich's ataxia (FA) in patients 16 years of age and older.2 This approval – granted under Health Canada's Priority Review process – marks an important milestone making SKYCLARYS the only treatment in Canada to specifically target the underlying mechanisms of this rare, progressive neurodegenerative disease.

Addressing an unmet need in Friedreich's ataxia
Before SKYCLARYS, individuals with Friedreich's ataxia faced a significant gap in care with no available treatments to slow the progression of the disease. This approval offers a meaningful option for eligible Canadians living with this life-altering and life-shortening disease3 who have long faced limited treatment options.

"Friedreich's ataxia presents profound physical challenges, progressively affecting motor function, independence, and overall quality of life. As a physician and researcher in this field, I have seen firsthand the critical need for treatments that address the underlying causes of this devastating disease," said renowned Montreal-based neurologist and researcher, Dr. Massimo Pandolfo, lead investigator in the international collaboration that identified the Friedreich's ataxia (FRDA) gene. "The approval of SKYCLARYS is a significant advancement, offering the first therapy to go beyond managing symptoms and to targeting disease progression. While not a cure, this development represents meaningful progress in treatment options and brings renewed hope to patients and their families."

Hope for a disease that impacts patients significantly
FA is a debilitating genetic condition affecting approximately 1 in 40,000 individuals in Canada4, with an estimated 300 to 750 people living with the disease.5 FA causes progressive damage to the spinal cord, peripheral nerves, and the brain regions that control balance, movement, and speech.6 Symptoms typically emerge in childhood or adolescence and worsen over time, leading to the loss of muscle coordination, speech and swallowing, and mobility, and complications such as vision and hearing impairment, scoliosis, diabetes, and an increased risk of serious heart conditions.7,8,9 The condition significantly impacts quality of life, with an average life expectancy of 37 years.9,10,11

"The approval of SKYCLARYS brings long-awaited hope to the patient community. It is a significant milestone for Canadians affected by Friedreich's ataxia and one our founder Claude St Jean would have been thrilled about. This therapy marks a crucial development in addressing a condition that severely impacts the lives of those living with it and their families," said François-Olivier Théberge, General Manager, Ataxia Canada. "For the first time, a treatment for this debilitating condition is within reach, bringing much needed optimism, and the potential for improved disease management."

The pivotal MOXIe Part 2 trial, a placebo-controlled study involving over 100 participants, formed the basis for Health Canada's approval of SKYCLARYS. After 48 weeks, patients treated with SKYCLARYS demonstrated a statistically significant improvement in modified Friedreich's Ataxia Rating Scale (mFARS) scores, highlighting its impact on disease progression.2

"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," said Eric Tse, General Manager of Biogen Canada. "We are incredibly proud to introduce the only Health Canada-approved treatment for Friedreich's ataxia. 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."

About SKYCLARYS™ (omaveloxolone)
SKYCLARYS™ (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich's ataxia (FA) in adults and adolescents aged 16 years and older in the United States (U.S.), European Union and Canada.2, 12,13 SKYCLARYS received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA).12 The European Commission granted Orphan Drug designation in Europe to SKYCLARYS for the treatment of FA.13 In Canada, marketing authorization for SKYCLARYS was granted under the Health Canada Priority Review process.14

Biogen continues to be dedicated to advancing research and development efforts to enhance the understanding of FA and improve patient outcomes. This includes an ongoing, open-label, Phase 1 study to evaluate the use of SKYCLARYS in in pediatric patients aged 2 to 15 years. More details can be found at clinicaltrials.gov.

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. Ataxia Canada. Friedreich's Ataxia. Available at: https://lacaf.org/en/ataxias/formes-and-transmission/friedreichs-ataxia. Accessed March 2025.
  2. SKYCLARYS™ Canadian Product Monograph. Biogen.ca/products/SKYCLARYS_PM_EN
  3. Friedrich's Ataxia Research Alliance. What is Friedreich's ataxia? Available at: https://www.curefa.org/understanding-fa/what-is-friedreichs-ataxia/. Accessed: March 2025.
  4. SickKids. Friedreich ataxia (FRDA). Available at: https://www.aboutkidshealth.ca/friedreich-ataxia-frda. Accessed March 2025.
  5. Polek B, Roach MJ, Andrews WT, Ehling M, Salek S. Burden of Friedreich's Ataxia to the Patients and Healthcare Systems in the United States and Canada. Front Pharmacol. 2013 May 22;4:66. doi: 10.3389/fphar.2013.00066. PMID: 23734128; PMCID: PMC3660667. 
  6. National Institute of Neurological Disorders and Stroke. Friedreich Ataxia. Available at: https://www.ninds.nih.gov/health-information/disorders/friedreich-ataxia. Accessed March 2025.
  7. Fogel BL, Perlman S. Clinical features and molecular genetics of autosomal recessive cerebellar ataxias. Lancet Neurol. 2007 Mar;6(3):245-57. doi: 10.1016/S1474-4422(07)70054-6. PMID: 17303531. 
  8. National Institute of Neurological Disorders and Stroke. Friedreich Ataxia. Available at: https://www.ninds.nih.gov/health-information/disorders/friedreich-ataxia. Accessed March 2025. 
  9. 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.
  10. 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. 
  11. 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.
  12. U.S. Food and Drug Administration. (2023, February 28) FDA approves first treatment for Friedreich's ataxia [press release] https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-friedreichs-ataxia. Accessed March 2025.
  13. Biogen Inc. (2024, February 12) Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich's Ataxia [press release] https://investors.biogen.com/news-releases/news-release-details/biogen-received-european-commission-approval-skyclarysr. Accessed March 2025.
  14. Health Canada. Priority Review of Drug Submissions (Therapeutic Products) Available at: https://www.canada.ca/content/dam/hc-sc/migration/hc-sc/dhp-mps/alt_formats/hpfb-dgpsa/pdf/prodpharma/prfs_tpfd-eng.pdf. Accessed March 2025.

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