Biogen's New Drug Submission for Omaveloxolone Accepted for Priority Review by Health Canada for the Treatment of Friedreich's Ataxia

• 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 Submission reflects Biogen's commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients

TORONTO, Sept. 17, 2024 /CNW/ - Biogen Canada Inc. (Biogen) is pleased to announce today that Health Canada has accepted for priority review its New Drug Submission (NDS) for omaveloxolone, an innovative therapy for Friedreich's ataxia for the treatment of adults and adolescents aged 16 years and older.

This once-daily oral medication, if approved, would represent the only therapy specifically targeted for Friedreich's ataxia, addressing a critical unmet treatment need in this patient population.

"Friedreich's ataxia 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."

Friedreich's ataxia is a rare, inherited condition that affects about 1 in 40,000 people and has a profound impact on their lives, causing a slow, progressive loss of muscle coordination, slurred speech, weakness, and sensory loss.2 Symptoms typically begin in childhood or adolescence, and while the progression varies, it often leads to significant disability over time2 with individuals becoming wheelchair-bound 8 to 10 years after disease onset.1

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

Omaveloxolone received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration, as well as Orphan Drug status by the European Commission.3,4 It has 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 Canada, priority review submissions follow an accelerated timeline where the standard review period is shortened to 180 days.5

A regulatory decision on the New Drug Submission for omaveloxolone is anticipated in early 2025.

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment, to deliver long-term growth.

Biogen has been proudly serving Canadian patients for more than 25 years. For information about Biogen Canada, please visit www.biogen.ca.

References:

1. Ataxia Canada. Friedreich's Ataxia. Available at: https://lacaf.org/en/ataxias/formes-and-transmission/friedreichs-ataxia. Accessed September 2024.
2. AboutKidsHealth. Friedreich ataxia. Jan. 2020. Available at: https://www.aboutkidshealth.ca/friedreich-ataxia-frda. Accessed September 2024.
3. U.S. Food and Drug Administration. (2023, February 28) FDA approves first treatment for Friedreich's ataxia [press release] https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-friedreichs-ataxia Accessed September 2024.
4. Biogen Inc. (2024, February 12) Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich's Ataxia [press release] https://investors.biogen.com/news-releases/news-release-details/biogen-received-european-commission-approval-skyclarysr Accessed September 2024.
5. Health Canada. Priority Review of Drug Submissions (Therapeutic Products) Available at: https://www.canada.ca/content/dam/hc-sc/migration/hc-sc/dhp-mps/alt_formats/hpfb-dgpsa/pdf/prodpharma/prfs_tpfd-eng.pdf Accessed September 2024.

SOURCE Biogen Canada Inc.