Friedreich’s ataxia treatment Skyclarys approved in Canada
Therapy is 1st disease-modifying FA treatment to be authorized in country
Biogen’s Skyclarys (omaveloxolone) has been approved in Canada to treat people with Friedreich’s ataxia (FA) ages 16 and older, becoming the first disease-modifying therapy for FA to receive regulatory clearance in the country.
The therapy was approved under Health Canada’s priority review process, which accelerates the review timeline for treatments that could fill certain unmet therapeutic needs.
Skyclarys has received similar approvals in the U.S., the European Union, and Switzerland. Biogen submitted an application to regulators in the U.K. last November.
“We are incredibly proud to introduce the only Health Canada-approved treatment for Friedreich’s ataxia,” Eric Tse, general manager of Biogen Canada, said in a company press release. “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.”
FA is a genetic condition in which low levels of the frataxin protein drive impairments in mitochondria, the structures responsible for producing cellular energy. Reduced energy production, inflammation, and a type of cellular damage called oxidative stress contribute to the degeneration of nerve cells involved in muscle control.
Friedreich’s ataxia treatment
A once-daily oral therapy, Skyclarys works to slow disease progression by activating nuclear factor erythroid 2-related factor 2, a protein that increases the activity of genes that promote mitochondrial function, block inflammation, and prevent oxidative stress.
Before Skyclarys became available to treat certain patients, FA management largely relied on treatments and interventions to ease symptoms and preserve function. No therapies specifically targeted underlying disease mechanisms to slow FA progression.
“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 Massimo Pandolfo, MD, leader of the international collaboration that identified the gene associated with FA in the 1990s.
“The approval of Skyclarys is a significant advancement, offering the first therapy to go beyond managing symptoms and to targeting disease progression,” said Pandolfo, co-director of the Montreal Neurological Institute and Hospital’s clinical research unit and professor at McGill University. Though Skyclarys is not a cure for FA, “this development represents meaningful progress in treatment options and brings renewed hope to patients and their families,” Pandolfo said.
The therapy’s efficacy in FA patients was evaluated in the two-part Phase 2 MOXIe clinical trial (NCT02255435) involving patients ages 16-40.
Data from the study’s second part largely supported the recent Canadian approval, according to Biogen. That part of the trial enrolled 103 participants, who were randomly assigned to receive daily Skyclarys (150 mg) or a placebo for 48 weeks, or nearly a year.
Skyclarys led to a significant, 2.4-point decrease in the scores of the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to the placebo after 48 weeks, reflecting an improvement in neurological function.
Results from the study’s open-label extension phase, in which all patients who completed the main MOXIe trial could receive Skyclarys, showed the benefits of the therapy were generally maintained over time, with rates of disease progression slower than those observed in the natural history of the disease.
Per the Canadian label, common side effects of Skyclarys include elevated liver enzymes, headache, nausea, fatigue, and diarrhea.
François-Olivier Théberge, general manager for Ataxia Canada, called the approval a “significant milestone for Canadians affected by Friedreich’s ataxia.”
“For the first time, a treatment for this debilitating condition is within reach, bringing much needed optimism, and the potential for improved disease management,” Théberge said.
All regulatory approvals for Skyclarys thus far cover FA patients ages 16 and older. An ongoing Phase 1 study called BOLD (NCT06054893) is evaluating how Skyclarys is processed in the body and how safe it is among pediatric FA patients ages 2-15. The trial is recruiting about 20 participants at a single site in Philadelphia.
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