US-based biotechnology company Biogen has secured Health Canada approval for Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA) in patients aged 16 years and above.
Skyclarys is a second-generation synthetic oleanane triterpenoid compound, already approved in the European Union (EU) and the US for the treatment of FA.
The current approval, granted under the Priority Review process, makes Skyclarys the first treatment in Canada to specifically target the underlying mechanisms of FA.
Before the approval, individuals with Friedreich’s ataxia in Canada had no treatment options to slow disease progression, creating a significant gap in care.
Biogen Canada general manager Eric Tse said: “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 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.”
The approval of Skyclarys was based on the Part 2 MOXIe clinical trial, a placebo-controlled study involving over 100 participants.
In the study, patients treated with Skyclarys showed a statistically significant improvement in modified FA Rating Scale (mFARS) scores at 48 weeks, impacting disease progression.
FA is a rare neurodegenerative disease that causes progressive damage to the spinal cord, peripheral nerves, and brain regions controlling balance, movement, and speech.
The symptoms typically emerge in childhood or adolescence and progress over time, leading to loss of muscle coordination, speech and swallowing difficulties, and mobility issues.
Its complications include vision and hearing impairment, scoliosis, diabetes, and an increased risk of serious heart conditions.
The Friedreich’s ataxia gene (FRDA) was identified by an international collaboration.
International collaboration lead investigator Massimo Pandolfo said: “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.
“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.”
