Health Canada has approved Biogen’s SKYCLARYS (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients 16 years of age and older. 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.
“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 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.”
FA is a debilitating genetic condition affecting approximately 1 in 40,000 individuals in Canada, with an estimated 300 to 750 people living with the disease. FA causes progressive damage to the spinal cord, peripheral nerves, and the brain regions that control balance, movement, and speech.
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. The condition significantly impacts quality of life, with an average life expectancy of 37 years.
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.
“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.”
