Health Canada Accepts Biogen's Omaveloxolone for Priority Review in Treating Friedreich's Ataxia

Biogen Canada Inc. announced that Health Canada has accepted its New Drug Submission (NDS) for priority review concerning omaveloxolone, a groundbreaking treatment for Friedreich’s ataxia. This therapy is intended for adults and adolescents aged 16 and older, promising to be the first disease-specific treatment for this progressive and debilitating neuromuscular condition.

Eric Tse, General Manager of Biogen Canada, emphasized the substantial impact Friedreich’s ataxia has on patients and their families, noting that the acceptance of the NDS for omaveloxolone represents a significant advancement in providing hope and potential relief for individuals suffering from this severe neurodegenerative disorder. Biogen is committed to collaborating with Canadian health authorities to make this promising treatment accessible to those in need.

Friedreich’s ataxia is a rare genetic disorder that affects approximately 1 in 40,000 people. It severely impacts patients' lives by causing a gradual loss of muscle coordination, slurred speech, muscle weakness, and sensory loss. Symptoms typically manifest in childhood or adolescence and worsen over time, often leading to significant disabilities, with many patients becoming wheelchair-bound within 8 to 10 years of onset.

The NDS for omaveloxolone is underpinned by efficacy and safety data derived from the placebo-controlled MOXIe Part 2 trial. This study demonstrated that omaveloxolone significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores over 48 weeks compared to a placebo.

Omaveloxolone has already received several designations from the U.S. Food and Drug Administration, including Orphan Drug, Fast Track, and Rare Pediatric Disease statuses. It has also been granted Orphan Drug status by the European Commission. The treatment has regulatory approval in both the United States and the European Union for patients aged 16 years and older, receiving robust support from both the medical community and patient advocacy groups.

Priority review submissions in Canada follow an accelerated timeline, reducing the standard review period to 180 days. A regulatory decision on the NDS for omaveloxolone is expected in early 2025.

Biogen, founded in 1978, is a leading biotechnology company dedicated to pioneering innovative science to develop new medicines that transform patient lives. The company leverages a deep understanding of human biology and employs various modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Biogen’s strategy involves taking bold yet balanced risks to ensure long-term growth while serving the unmet needs of patients. The company has been serving Canadian patients for over 25 years, demonstrating a strong commitment to improving lives through innovative healthcare solutions.