Health Canada Grants Priority Review to Biogen's Omaveloxolone for Friedreich's Ataxia
Key Insights
Health Canada has accepted Biogen's New Drug Submission for omaveloxolone under priority review for treating Friedreich's ataxia in patients aged 16 and older.
Omaveloxolone, a once-daily oral medication, could become the first disease-specific treatment option available for this rare and debilitating neuromuscular condition.
The submission is supported by data from the MOXIe Part 2 trial, which demonstrated significant improvements in mFARS scores compared to placebo at 48 weeks.
Biogen Canada Inc. announced that Health Canada has accepted its New Drug Submission (NDS) for omaveloxolone for priority review. Omaveloxolone is an investigational, innovative therapy intended for the treatment of Friedreich’s ataxia (search) in adult and adolescent patients aged 16 years and older. If approved, omaveloxolone would be the first and only treatment specifically for Friedreich's ataxia, addressing a significant unmet need for Canadian patients.
Addressing a Critical Unmet Need
Friedreich’s ataxia (search) is a rare, inherited disease affecting approximately 1 in 40,000 individuals. This condition leads to progressive loss of muscle coordination, slurred speech, weakness, and sensory loss. Symptoms typically manifest during childhood or adolescence, often resulting in substantial disability, with many individuals becoming wheelchair-bound within 8 to 10 years of disease onset.
"Friedreich’s ataxia (search) is a devastating diagnosis that profoundly impacts the lives of patients and their families," said Eric Tse, General Manager at Biogen Canada. "The omaveloxolone NDS acceptance marks a significant step forward for Canadian patients, offering hope and potential for a therapy that could help manage this debilitating neurodegenerative disorder. We look forward to working with Canadian health authorities to bring forward this promising treatment for those in need."
Supporting Clinical Data
The NDS for omaveloxolone is backed by efficacy and safety results from the MOXIe Part 2 trial, a placebo-controlled study. The data indicated that omaveloxolone led to statistically significant improvements in modified Friedreich Ataxia Rating Scale (mFARS) scores at 48 weeks compared to placebo.
Regulatory Status and Timeline
Omaveloxolone has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the U.S. Food and Drug Administration, and Orphan Drug status by the European Commission. It has already received regulatory approval for treating Friedreich’s ataxia (search) in adults and adolescents aged 16 and older in both the United States and the European Union.
Health Canada’s priority review process accelerates the standard review timeline to 180 days. A regulatory decision regarding the approval of omaveloxolone in Canada is expected in early 2025.