Health Canada accepts Biogen's new drug application for omaveloxolone for the treatment of Friedreich's ataxia under priority review

• Friedreich's ataxia is a rare, debilitating and life-shortening neuromuscular disease caused by genetic factors¹
• If approved, omaveloxolone would be the only disease-specific treatment option for patients living with this progressive and challenging disease.
• This New Drug Application reflects Biogen's commitment to advancing treatments for rare diseases and meeting the unmet needs of Canadian patients.

TORONTO, 17 September 2024 /CNW/ – Biogen Canada Inc. (Biogen) is pleased to announce that Health Canada has accepted for priority review the New Drug Submission (NDS) for omaveloxolone, an innovative therapeutic for Friedreich’s ataxia for the treatment of adults and adolescents 16 years of age and older.

If approved, this once-daily oral medication would be the only therapy specifically targeting Friedreich's ataxia, addressing a critical unmet treatment need in this patient group.

“Friedreich’s ataxia is a devastating diagnosis that has a profound impact on the lives of patients and their families,” said Eric TseGeneral Manager at Biogen Canada. “The NDS approval for omaveloxolone is a significant step forward for Canadian patients and offers hope and potential for a therapy that could help treat this debilitating neurodegenerative disease. We look forward to working with Canadian health authorities to advance this promising treatment for those in need.”

Friedreich's ataxia is a rare inherited disease that affects about 1 in 40,000 people and has a profound impact on their lives. It causes a slow, progressive loss of muscle coordination, slurred speech, weakness and sensory loss.² Symptoms typically begin in childhood or adolescence, and although the course varies, over time this often leads to significant disability² Eight to ten years after the onset of the disease, those affected are confined to a wheelchair.¹

The NDS for omaveloxolone is supported by efficacy and safety data from the placebo-controlled MOXIe Part 2 study, which found that omaveloxolone significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores at 48 weeks compared with placebo.

Omaveloxolone has been granted orphan drug, fast track medicine, and rare pediatric disease designation by the U.S. Food and Drug Administration and orphan drug designation by the European Commission.^3.4 It received regulatory approval for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union, with strong support from the medical and patient communities.^3.4

In CanadaSubmissions for priority review follow an expedited schedule, with the standard review period reduced to 180 days.⁵

A regulatory decision on the marketing authorization application for omaveloxolone is expected in early 2025.