Omaveloxolone

2025年6月18日,Biogen公司宣布启动全球III期临床试验BRAVE研究的给药工作。该研究旨在评估Omaveloxolone对2至16岁以下弗里德赖希共济失调（FA）患儿的疗效和安全性。无论是否具备行走能力，患者均可能符合入组条件。受试者将以2:1的比例随机分配，每日接受一次Omaveloxolone或安慰剂治疗52周，随后可转入开放标签扩展期（OLE）。目前，Omaveloxolone以品牌名SKYCLARYS®在40多个国家（包括美国和欧盟）上市，是唯一获批用于16岁及以上青少年和成人FA患者的药物。关键研究设计BRAVE研究计划纳入约255名FA患儿，评估Omaveloxolone的疗效、安全性、药代动力学和药效学。研究分为两部分：第1阶段：为期52周的双盲、随机、安慰剂对照试验，主要终点为直立稳定性评分（USS）较基线的变化。USS是经FA社群认可的、用于评估儿童疾病进展最敏感的指标，隶属于验证版改良FA评定量表（mFARS）。第2阶段：开放标签扩展期（最长至104周），所有受试者将接受Omaveloxolone治疗，以进一步评估长期效果。专家观点Biogen神经肌肉开发单元负责人Stephanie Fradette博士："弗里德赖希共济失调症状通常始于儿童期，且发病越早，疾病进展越快。儿科患者存在巨大未满足需求。基于Reata的研究基础，我们正加速推进Omaveloxolone的儿科开发计划。"加州大学洛杉矶分校共济失调中心主任Susan Perlman教授："早发型患者往往病情进展迅猛，而目前尚无获批疗法。BRAVE研究将评估Omaveloxolone对这一脆弱人群的潜在获益。"关于SKYCLARYS®（Omaveloxolone）SKYCLARYS®是一种每日一次的口服药物，已在40余个国家（含美欧）获批用于16岁及以上FA患者。该药曾获美国FDA授予的孤儿药、快速通道和罕见儿科疾病认定，以及欧盟的孤儿药资格。安全信息：完整处方信息请参见美国产品说明书或各国注册网站。关于弗里德赖希共济失调（FA）FA是一种罕见的遗传性神经肌肉退行性疾病，为最常见的遗传性共济失调类型。其特征包括：早期症状：协调能力丧失、肌无力、疲劳（常于儿童期出现）疾病进展：多数患者在首发症状后10-20年内需依赖轮椅生存现状：报告显示患者平均寿命仅37岁，但通过针对性护理可显著延长生存期药事纵横投稿须知：稿费已上调，欢迎投稿\

Global Phase 3 BRAVE study will evaluate the efficacy and safety of omaveloxolone in children 2 to <16 years old with Friedreich ataxia, a rare neurodegenerative disorder BRAVE study will explore the potential of omaveloxolone to address the critical unmet need of the pediatric FA population Omaveloxolone is currently marketed under the brand name SKYCLARYS® for the treatment of adults and adolescents aged 16 years and older affected by Friedreich ataxia CAMBRIDGE, Mass., June 18, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced the initiation of dosing in the BRAVE study, a global Phase 3 clinical trial. The BRAVE study will evaluate the efficacy and safety of omaveloxolone in children with Friedreich ataxia (FA) between the ages of 2 to <16. Both non-ambulatory and ambulatory participants may qualify for the study. Participants will be randomized 2:1 to receive omaveloxolone or placebo once a day for 52 weeks before having the opportunity to move into the open-label extension (OLE). Currently, omaveloxolone is commercialized under the brand name SKYCLARYS® in over 40 countries, including in the U.S. and the European Union, and is the only approved product for FA in adults and adolescents aged 16 years and older. “Recognizing the symptoms of Friedreich ataxia typically begin in childhood, and earlier onset of symptoms is associated with faster disease progression, there is tremendous unmet need in the pediatric community. Building on the work of Reata we have been urgently advancing the pediatric development plan for omaveloxolone and are thrilled that the Phase 3 BRAVE study has now begun,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. “We are immensely grateful for the input from the entire FA community that has helped shape the design of this important study.” The BRAVE study will evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of omaveloxolone in approximately 255 children living with FA. Part 1 is a 52 week, randomized, double-blind, placebo-controlled study, designed to evaluate efficacy of omaveloxolone compared to placebo. The primary outcome measure for Part 1 is change from baseline in Upright Stability Score (USS), a subscale that is recognized by the FA community as the most sensitive means of measuring disease progression in children living with FA. USS is part of the validated modified FA rating scale (mFARS). Part 2 will be an open-label extension (up to week 104) where all participants will receive omaveloxolone to further our understanding of the long-term effects of the drug. “Early onset patients often have the most aggressive and fast progressive form of Friedreich ataxia and through the BRAVE study we aim to determine the potential safety and efficacy of omaveloxolone for children living with the disease. This vulnerable population faces significant unmet need, with no approved treatments currently available,” said Susan Perlman, M.D., Professor of Neurology and Director of the Ataxia Center, David Geffen School of Medicine at UCLA. The design of this Phase 3 study has been informed by previous studies and input from investigators, global medical experts and the FA community. Enrollment has initiated in the United States and we plan to open BRAVE study sites around the world pending final alignment with local regulators and ethics committees. Individuals interested in participating in this study should speak with their healthcare provider. They can also email Biogen at clinicaltrials@biogen.com. Individuals located in the U.S. can also call the Biogen Clinical Trials Center at 866-633-4636. Both clinicaltrials.gov (NCT06953583) and Biogen Trial Link (Biogen Trial Link) will be updated as more information about the BRAVE study becomes available. About SKYCLARYS® (omaveloxolone)SKYCLARYS® (omaveloxolone) is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older in over 40 countries, including the U.S. and European Union. SKYCLARYS received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration. The European Commission granted Orphan Drug designation in Europe to SKYCLARYS for the treatment of FA. Please click here for Important Safety Information and full Prescribing Information for SKYCLARYS® (omaveloxolone) in the U.S. or visit your respective country’s product website. About Friedreich AtaxiaFriedreich ataxia (FA) is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder. It is the most common inherited ataxia.1,2,3 Early symptoms of FA, such as progressive loss of coordination, muscle weakness and fatigue, typically appear in childhood and can overlap with other diseases.4 Most people living with FA will need to use a wheelchair within 10-20 years of their first symptoms.2 The reported average age of death for FA patients is just 37 years old, although with appropriate and targeted care, individuals may live many years after confinement to a wheelchair.5-7 About BiogenFounded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ 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.We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube. Biogen Safe Harbor This news release contains forward-looking statements including relating to the potential benefits, safety and efficacy of SKYCLARYS; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of Friedreich’s ataxia; the potential of Biogen’s commercial business and pipeline programs, including Friedreich’s ataxia; and risks and uncertainties associated with drug development and commercialization. These statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would” and other words and terms of similar meaning. 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