Omaveloxolone

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. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, factors relating to: our substantial dependence on revenue from our products and other payments under licensing, collaboration, acquisition or divestiture agreements; uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the drivers for growing our business; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential future healthcare reforms; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third parties, intellectual property, competitive and market challenges and regulatory compliance; failure to obtain, protect, and enforce our data, intellectual property, and other proprietary rights and the risks and uncertainties relating to intellectual property claims and challenges; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; risks relating to technology, including our incorporation of new technologies such as artificial intelligence into some of our processes; risks related to use of information technology systems and potential impacts of any breakdowns, interruptions, invasions, corruptions, data breaches, destructions and/or other cybersecurity incidents of our systems or those of connected and/or third-party systems; problems with our manufacturing capacity, including our ability to manufacture products efficiently or adequately address global bulk supply risks; risks relating to management, personnel and other organizational changes, including our ability to attracting, retaining and motivating qualified individuals; risks related to the failure to comply with current and new legal and regulatory requirements, including judicial decisions, accounting standards, and tariff or trade restrictions; the risks of doing business internationally, including geopolitical tensions, acts of war and largescale crises; risks relating to investment in our manufacturing capacity; risks relating to the distribution and sale by third parties of counterfeit or unfit versions of our products; risks relating to the use of social media for our business, results of operations and financial condition; fluctuations in our operating results; risks related to investment in properties; risks relating to access to capital and credit markets to finance our present and future operations and business initiatives and obtain funding for such activities on favorable terms; risks related to indebtedness; the market, interest, and credit risks associated with our investment portfolio; risks relating to share repurchase programs; change in control provisions in certain of our collaboration agreements; fluctuations in our effective tax rate and obligations in various jurisdictions in which we are subject to taxation; environmental risks; and any other risks and and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned “Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. References:

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period. LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Friedreich's Ataxia Market Report According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034. The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals. Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing. Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years. The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others. Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report Friedreich's Ataxia Market Dynamics The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology. The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification. Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM. However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies. Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth. Friedreich's Ataxia Treatment Market Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected. The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective. Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles. Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation. SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects. It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone. To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication Friedreich's Ataxia Pipeline Therapies and Key Companies Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others. Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025. RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021. Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition. The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials Recent Developments in the Friedreich's Ataxia Market In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression. In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review. In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia. Friedreich's Ataxia Overview Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination. The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease. In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech. Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis. Friedreich's Ataxia Epidemiology Segmentation The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalent Cases of Friedreich's Ataxia Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia Total Treated Cases of Friedreich's Ataxia Scope of the Friedreich's Ataxia Market Report Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market Table of Contents Related Reports Friedreich's Ataxia Epidemiology Forecast Friedreich's Ataxia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Friedreich's ataxia epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Friedreich's Ataxia Pipeline Friedreich's Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Friedreich's ataxia companies, including PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, among others. Ataxia Market Ataxia Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key ataxia companies, including AstraZeneca, Merck Sharp & Dohme LLC, Bayer, Roche, among others. Ataxia Pipeline Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ataxia companies, including Retrotope, Reata Pharmaceuticals, PTC Therapeutics, Metro International Biotech, LLC, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, EryDel, Biogen, Matrix Biomed, IntraBio, Biohaven Pharmaceuticals, Inc., Stealth BioTherapeutics Inc., Acasti Pharma, Seelos Therapeutics, Kissei Pharmaceutical, Vico Therapeutics, Q-State Biosciences, Locanabio, Lexeo Therapeutics, Voyager Therapeutics, CRISPR Therapeutics, Capsida Biotherapeutics, AavantiBio, StrideBio, Wave Life Sciences, REPROCELL, SHIONOGI & Co., CORESTEM, Blade Therapeutics, Exicure, Lacerta Therapeutics, Healx Ltd., Uniqure, Ionis Pharmaceuticals, Jupiter Neurosciences, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED