Ataxia treatment Skyclarys closer to reimbursement in Quebec
Biogen says agency's re-evaluation of data puts therapy on path
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Biogen’s Skyclarys (omaveloxolone) is one step closer to being covered by Quebec public healthcare systems for people with Friedreich’s ataxia (FA), ages 16 and older, with the province’s Institut national d’excellence en santé et en services sociaux’s (INESSS) re-evaluation of the therapy after initially declining to recommend it.
Skyclarys was approved in Canada in March 2025 under Health Canada’s priority review process, which speeds the review of treatments that could fill certain unmet therapeutic needs. This was followed by a positive reimbursement recommendation for Skyclarys from Canada’s Drug Agency in July 2025.
INESS, after reviewing four years of data, has now concluded Skyclarys could have some benefit for patients.
“We are encouraged by INESSS’s recognition of the therapeutic value of SKYCLARYS and the urgent unmet need within the Friedreich ataxia community,” Eric Tse, general manager of Biogen Canada, said in a company press release. “We remain committed to working with governments, clinicians, and the patient community to help improve access for eligible people across Canada as quickly as possible.”
François-Olivier Théberge, general manager of Ataxia Canada, said: “Given the comparatively higher number of individuals affected by Friedreich’s ataxia in Québec, recognition of the therapeutic value of SKYCLARYS and movement toward public reimbursement in the province marks an important milestone for individuals living with this progressive disease, for whom timely access to treatment is critical.”
Second evaluation looks at new data
FA is a progressive, hereditary neuromuscular disease characterized by impaired balance and coordination that leads to loss of mobility and a significant decline in quality of life. Other FA symptoms can include fatigue, speech difficulties, heart problems, scoliosis (sideways curvature of the spine), and diabetes.
FA care primarily focuses on managing symptoms and treating heart complications and diabetes, but none of this slows the progression of the disease. Mobility aids, wheelchairs, and adaptive clothing help patients maintain some independence.
“For individuals living with this relentlessly progressive disease, maintaining mobility and independence for longer can have a profound impact on quality of life,” said Antoine Duquette, MD, a Montreal-based neurologist specializing in FA and inherited neurological disorders.”
Skyclarys slows disease progression by suppressing the inflammation associated with FA and reducing oxidative stress, a type of cellular damage.
The therapy’s approval in Canada was based on data from the MOXIe Phase 2 clinical trial (NCT02255435). In the study’s open-label extension phase, participants treated with Skyclarys for nearly three years had slower disease progression than observed in the natural history of the disease.
“After years without approved treatment options for Friedreich ataxia, the recognition of a therapy that may help slow disease progression and help preserve function represents a significant development for clinicians and the FA community,” Duquette said. “Progress toward bringing treatment within reach for eligible patients in Québec is therefore especially significant.”
This was INESSS’ second FA evaluation of Skyclarys. The first evaluation was based on MOXIe data, along with extension-phase data through 72 weeks (about 1.4 years). At that time, INESSS found that the difference of about 2 points on the modified Friedreich’s Ataxia Rating Scale (mFARS), a validated measure of neurological function, was modest.
Moreover, no benefit was demonstrated on validated measures of quality of life or maintenance of independence, and long-term effectiveness data were considered uncertain. INESSS concluded that it was not possible to determine whether Skyclarys could meet the health needs of FA patients.
For this second evaluation, new data were considered, including up to four years of extension-phase data confirming slower disease progression and functional decline than the natural course of the disease. And results in specific areas, particularly speech and upper-limb coordination, suggested stability over time that, in the view of the consulted clinicians, could translate into a real clinical benefit for some patients. Families also reported that small improvements have real value in daily life.
“For individuals living with this relentlessly progressive disease, maintaining mobility and independence for longer can have a profound impact on quality of life,” Duquette said.
Based on these new data, INESSS concluded that Skyclarys could partly meet the health needs of FA patients.
“For the Friedreich ataxia community, this outcome represents an encouraging and meaningful development,” Théberge said. “People living with FA and their families continue to face significant physical, emotional, and daily challenges associated with this progressive disease.”
Despite recognizing a partial clinical benefit, INESSS reiterated that Skyclarys’ cost is too high and needs to come down for reimbursement. That is, the additional cost compared to best supportive care alone could not be justified by its clinical benefits, and the cost-effectiveness ratio was deemed unfavorable.
INESSS estimated that during the first three years, coverage of Skyclarys would generate approximately $79.5 million in additional spending on Quebec’s public drug insurance plan budget for the treatment of an estimated 141 patients.
