Omaveloxolone

Friedreich’s ataxia is a rare, debilitating and life-shortening neuromuscular disease caused by genetic factors 1 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 time 2 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 . References: Ataxia Canada . Friedreich’s Ataxia . Available at: Accessed September 2024 . AboutKidsHealth. Friedreich ataxia . Jan. 2020 . Available at: . Accessed September 2024 . U.S. Food and Drug Administration. (2023, February 28 ) FDA approves first treatment for Friedreich’s ataxia [press release] Accessed September 2024 . Biogen Inc. (2024, February 12 ) Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia [press release] Accessed September 2024 . Health Canada . Priority Review of Drug Submissions (Therapeutic Products) Available at: Accessed September 2024 . SOURCE Biogen Canada Inc.

The biopharmaceutical landscape is quietly undergoing a transformation, with neuroscience emerging as a hotspot amidst the industry's shifting dynamics. While the cancer therapy arena has seen setbacks with dual antibodies and ADCs, the metabolic disease space, led by weight loss treatments, has experienced a surge in popularity. Concurrently, the neuroscience (CNS) field is witnessing a renaissance, marked by clinical breakthroughs and a series of high-profile mergers and acquisitions.   The renewed focus on neuroscience is driven by the immense unmet medical needs and recent advancements in fundamental research. According to WHO projections, neurological disorders are poised to become the second leading cause of death within the next 20 years. Alarmingly, the number of individuals diagnosed with Alzheimer's disease (AD) and other forms of dementia continues to rise, with an estimated 50 million people worldwide affected. Additionally, the prevalence of depression and severe mental illnesses is on the rise, with a worrying trend towards younger populations.   Despite this growing burden, the efficacy of existing treatment options for neurological disorders remains below 50%, presenting a vast clinical demand and market potential. In this landscape, even minor therapeutic differences can lead to commercial success, as "you don't need a cure to have a blockbuster, just an incremental improvement." Moreover, the rapid advancements in sequencing technologies and the resulting drop in costs have enabled exponential growth in genetic research related to neurodegenerative diseases. By analyzing relevant genes, scientists have been able to identify critical signaling pathways and drug targets.   The approval of Biogen's Aduhelm, the first Alzheimer's drug targeting the Aβ cascade, has served as a catalyst for the resurgence of the CNS field. Although Aduhelm faced challenges due to its significant side effects, leading to commercial failure, the recent full approval of Eisai and Biogen's Leqembi (lecanemab) in the United States and China marks a significant milestone. Leqembi is the first drug capable of slowing the progression of early-stage Alzheimer's disease, a landmark achievement in this notoriously difficult-to-treat field.   Sensing the renewed promise of neuroscience, multinational corporations (MNCs) have accelerated their efforts to establish a presence in this space. However, the shadows of past failures have made them more cautious, leading them to favor low-risk, late-stage transactions that avoid the risks associated with early and mid-stage clinical trials. In 2022, Pfizer acquired Biohaven for $11.6 billion, gaining access to the migraine drug Nurtec ODT (rimegepant). In 2023, Pfizer's CGRP receptor antagonist nasal spray, Zavzpret, successfully obtained FDA approval.   The neuroscience field has witnessed three major acquisition deals in 2023: Bristol Myers Squibb's $14 billion acquisition of Karuna Therapeutics, which provided the company with the schizophrenia drug KarXT; AbbVie's $8.7 billion purchase of Cerevel, securing the Parkinson's disease candidate Tavapadon and the schizophrenia and Alzheimer's psychosis candidate Emraclidine; and Biogen's $7.3 billion acquisition of Reata, gaining access to the Nrf2 activator Omaveloxolone for neurological indications.   As the industry navigates the complexities of the neuroscience landscape, the promise of genetic research, advancements in AI-driven precision medicine, and the recent regulatory successes have ignited a renewed sense of optimism. The stage is set for MNCs to continue their aggressive pursuit of promising neuroscience assets, driven by the urgent need to address the growing burden of neurological disorders and the potential for significant commercial rewards.