RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Australia), Priority Review (Canada), Rare Pediatric Disease (United States)

Structure/Sequence

Molecular FormulaC33H44F2N2O3

InChIKeyRJCWBNBKOKFWNY-IDPLTSGASA-N

CAS Registry1474034-05-3

Clinical Trials associated with Omaveloxolone

NCT06628687

/ RecruitingNot Applicable

A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications, and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and/or Lactation

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and/or while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the "SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program."
The main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life.
The main question researchers want to answer in this study is:
· Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects?
Researchers will also learn more about:
* Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects?
* Does taking BIIB141 during pregnancy or breastfeeding affect the following:
* Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar
* Pre-eclampsia, a pregnancy-related high blood pressure disease
* Unborn baby being small for its expected age (usually in weeks)
* Loss of an unborn baby
* Live birth
* Premature birth
* Loss of a newborn
* Growth or developmental delays in the baby
* Serious illness in the baby resulting in hospitalization
* Serious infections in the baby, or ones in babies with a weakened immune system
This study will be done as follows:
* Participants will join the study after signing an informed consent form, also known as an ICF.
* During the study, health information from the participants' regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born.
* Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.

Start Date26 Oct 2026

Sponsor / Collaborator

Biogen, Inc.

NCT07019064

/ RecruitingPhase 1

A Phase 1, Randomized, Open-label, Single-dose, Crossover, Relative Bioavailability Study of Omaveloxolone Intact Capsules Versus Capsule Contents Sprinkled Over Low-fat, Non-Greek Yogurt in Healthy Adult Subjects

In this study, researchers will learn more about how BIIB141, also known as omaveloxolone or SKYCLARYS?, is processed in the body when taken in different ways. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. Currently, people with FA can take it either as whole capsules or opening the capsules and sprinkling its contents over applesauce. The main goal of this study is to learn if BIIB141 is processed any differently when taken sprinkled over low-fat, non-Greek yogurt compared to whole capsules. This will help researchers learn if yogurt could be another option for people to take BIIB141 with.
The main questions researchers want to answer in this study are :
? How does the body process BIIB141 when taken as whole capsules compared to being sprinkled over yogurt?
Researchers will also learn more about :
* How many participants have adverse reactions during the study. An adverse reaction is a health problem that may be caused by the study drug.
* If there are any changes in the participants' overall health during the study
This study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.
* Participants will stay at the study research center for about 30 days.
* This is a "crossover" study. In this kind of study, all participants receive the same 2 or more study drugs (or ways of taking the drug), but the order in which they receive them depends on the group they are randomly assigned to.
* In Group 1, participants will take a single dose of BIIB141 as a capsule, followed by a break of 14 days. Then, the participants will take a single dose of BIIB141 sprinkled over yogurt.
* In Group 2, participants will take a single dose of BIIB141 sprinkled over yogurt, followed by a break of 14 days. Then, the participants will take a single dose of BIIB141 as a capsule.
* Each participant will be in the study for up to 57 days.

Start Date11 Jun 2025

Sponsor / Collaborator

Biogen, Inc.

NCT06953583

/ RecruitingPhase 3

A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS?. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old.
The main questions researchers want to answer in this study are:
* How does BIIB141 affect the participants' FA symptoms balance and stability?
* How many participants have medical problems during the study?
* Are there any changes in the participants' overall health during the study?
* Are there any changes in the participants' heart health?
* Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult.
Researchers will also learn more about:
- How the body processes BIIB141 in children and teens
This study will be done as follows:
* Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center.
* There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day.
* In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks.
* During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks.
* In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose.
* Each participant will be in the study for up to about 3 years

Start Date09 Jun 2025

Sponsor / Collaborator

Biogen, Inc.

100 Clinical Results associated with Omaveloxolone

100 Translational Medicine associated with Omaveloxolone

100 Patents (Medical) associated with Omaveloxolone

140

Literatures (Medical) associated with Omaveloxolone

01 Oct 2025·FREE RADICAL BIOLOGY AND MEDICINE

RTA-408 overcomes cisplatin-resistant lung cancer by inhibiting WWP1-mediated NCOA4 ubiquitination to induce ferritinophagy and ferroptosis

Article

Author: Xu, Ying ; Fei, Yingdan ; Yang, Yueyan ; Zhang, Sunhan ; Zhang, Ting ; Wu, Dongming ; Liu, Teng ; Deng, Shihua ; Wang, Xiaobian ; Chen, Zixin ; Zhu, Ruixiong

Worldwide, pulmonary carcinoma remains the predominant cause of cancer-induced fatalities. Cisplatin (DDP) is a platinum-containing cell cycle-non-specific chemotherapeutic agent and serves as the first-line treatment for non-small cell lung cancer (NSCLC). However, NSCLC cells gradually develop resistance to cisplatin during treatment, reducing drug sensitivity and leading to a significant decrease in the effect of chemotherapy or even treatment failure. The main mechanisms of drug resistance include reduced cellular uptake and apoptotic escape. Strategies to reverse drug resistance often suffer from off-target toxicity or inconsistent efficacy, highlighting the urgent need for new drugs that specifically target resistance pathways. Accordingly, we screened a library of small-molecule compounds associated with ferroptosis. We identified 4-7D (also known as RTA-408), a nuclear factor erythroid 2-related factor 2 activator initially developed to treat Friedreich's ataxia, which has shown potent anti-NSCLC activity in preclinical studies. Its unique ability to regulate both oxidative stress and apoptotic pathways makes it a promising candidate for overcoming cisplatin resistance. Therefore, the present study aimed to determine the key role of omaveloxolone (RTA-408) in overcoming cisplatin-resistant lung cancer. We selected A549/DDP cells as a research model and demonstrated through a series of experimental manipulations that RTA-408 inhibited the growth of A549/DDP cells and inhibited the ubiquitinated degradation of nuclear receptor coactivator 4 through down-regulation of WW structural domain-containing E3 ubiquitin-protein ligase 1 (WWP1), which further induced the development of ferritinophagy and ferroptosis. We report for the first time that RTA-408 overcomes cisplatin-resistant lung cancer by targeting WWP1-mediated ferritinophagy and ferroptosis.

01 Aug 2025·NEUROBIOLOGY OF DISEASE

Calcium-iron crosstalk in epileptogenesis: Unraveling mechanisms and therapeutic opportunities

Review

Author: Xiao, Fen ; Wang, Jing ; Wu, Nayiyuan ; Wang, Zhi-Bin ; Tao, Ao-Long ; Li, Xuan ; Zhang, Xiu

Epilepsy, a chronic neurological disorder affecting millions globally, remains poorly understood in its etiology and therapeutic management. Emerging evidence highlights the intricate interplay between calcium (Ca²⁺) and iron (Fe²⁺/Fe³⁺) ions in modulating neuronal excitability, oxidative stress, and synaptic plasticity-key processes implicated in epileptogenesis. This review synthesizes current knowledge on the dual roles of Ca²⁺ and Fe²⁺/Fe³⁺ in epilepsy, emphasizing their bidirectional regulatory mechanisms and pathological synergism. Calcium dysregulation, mediated through voltage-gated channels (e.g., Cav1.2, Cav3.2), store-operated calcium entry (SOCE), and mitochondrial calcium uniporters (MCU), exacerbates neuronal hyperexcitability and seizure propagation. Concurrently, iron overload drives ferroptosis via lipid peroxidation and glutathione depletion, while iron deficiency impairs neurodevelopmental processes. Crucially, Ca²⁺-Fe²⁺ crosstalk intersects at multiple nodes: TRP channels (e.g., TRPC6, TRPML1) facilitate dual ion transport; mitochondrial dysfunction links Ca²⁺ overload with Fe²⁺-dependent ROS generation; and inflammatory cascades disrupt both ion homeostasis. Clinically, antiepileptic drugs targeting Ca²⁺ channels (e.g., ethosuximide, zonisamide) and emerging ferroptosis inhibitors (e.g., deferoxamine, RTA 408) underscore the therapeutic potential of modulating these pathways. However, drug resistance and incomplete seizure control necessitate novel strategies leveraging ion interaction networks. We propose that combinatorial approaches targeting Ca²⁺-Fe²⁺ signaling hubs-such as MCU-TRPML1 axes or redox-sensitive RyR channels-may offer synergistic benefits. Future research must prioritize cross-model validation, advanced neuroimaging biomarkers, and multidisciplinary frameworks to translate mechanistic insights into precision therapies. This comprehensive analysis positions Ca²⁺-Fe²⁺ crosstalk as a pivotal frontier in epilepsy research, bridging molecular pathophysiology with innovative treatment paradigms.

01 Aug 2025·ECOTOXICOLOGY AND ENVIRONMENTAL SAFETY

Effect of the Nrf2/HO-1 pathway on aluminum-induced liver injury

Article

Author: Li, Dong ; Wei, Xi ; Luo, Yueling ; Wu, Biaoliang ; Qin, Xiaojie ; Yuan, Dongjie ; Nong, Yixi

Chronic exposure to aluminum (Al) can lead to the accumulation of Al in the liver, which negatively impacts liver structure and function. Oxidative stress is extensively identified as a key contributor to Al toxicity. The Nrf2/HO-1 pathway maintains cellular redox homeostasis and protects from oxidative stress through its antioxidative and cytoprotective role. However, its role in Al-induced liver damage remains unclear. In this study, rats were grouped into the control (C), Al exposure (Al), Al exposure + Omaveloxolone (Al+O), and Al exposure + ML385 (Al+M) groups, and their liver function, hepatic Al content, histopathological changes in liver tissue, and hepatocyte apoptosis were assessed. The Nrf2/HO-1 signaling- and apoptosis-associated protein levels were evaluated using Western blotting. In vitro, with BRL3A cells as a model, Al exposure's influence in cell viability, MMP, and Nrf2/HO-1- and apoptosis-associated protein levels was assessed. The Nrf2/HO-1 pathway was also modulated to investigate changes in oxidative stress and apoptosis-related markers following intervention. The results showed that Al accumulation in liver tissue led to rat histopathological changes and liver dysfunction, promoting oxidative stress and hepatocyte apoptosis, while also enhancing Nrf2/HO-1-associated protein levels. Similar findings were observed in in-vitro experiments. Furthermore, the Nrf2/HO-1 signaling activation mitigated Al-triggered oxidative stress and apoptosis, whereas its inhibition reduced these protective effects. This study indicated that Al exposure could induce s oxidative damage in rat livers and BRL3A cells; activation of the Nrf2/HO-1 pathway serves as a critical compensatory mechanism against Al toxicity, but its protective efficacy is limited.

News (Medical) associated with Omaveloxolone

18 Jun 2025

·investors.biogen.com

Biogen Initiates Phase 3 Pediatric Study of Omaveloxolone for the Treatment of Friedreich Ataxia

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:

Phase 3Orphan DrugFast TrackDrug Approval

05 Jun 2025

·www.prnewswire.com

Friedreich's Ataxia Market Poised for Rapid Expansion During the Forecast Period (2025-2034) as Drug Pipeline Gains Momentum | DelveInsight

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

Phase 3Orphan DrugFast TrackDrug ApprovalPhase 2

24 Apr 2025

·pmlive.com

Biogen’s Skyclarys granted MHRA approval for rare movement disorder Friedreich’s ataxia

Biogen’s Skyclarys (omaveloxolone) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as the first treatment for patients aged 16 years and older with the rare neurodegenerative movement disorder Friedreich’s ataxia (FA). FA is the most common inherited ataxia, a group of disorders affecting coordination, balance and speech, affecting at least one in every 50,000 people in the UK. Early symptoms of the life-shortening condition, such as progressive loss of coordination and muscle weakness, typically present in childhood and most patients will need to use a wheelchair within ten to 20 years of their first symptoms. Patients may also experience hearing loss, vision impairments, problems with speech and swallowing, scoliosis, diabetes and serious heart conditions as the disease progresses. Taken as an oral capsule, Skyclarys activates the Nrf2 pathway, which helps cells respond to a condition that can lead to cell and tissue damage. The MHRA’s decision on the drug was supported by positive results from the placebo-controlled MOXIe trial, in which Skyclarys-treated patients scored better on tests of neurological function compared to those in the placebo cohort. As previously reported by Biogen, all components of the modified FA Rating Scale (mFARS) assessment, including the ability to swallow, upper limb coordination, lower limb coordination and upright stability, favoured Skyclarys over placebo. Julian Beach, MHRA interim executive director of healthcare quality and access, said: “I am pleased to confirm the first UK approval for the treatment of FA, [Skyclarys]. “We’re assured that the appropriate regulatory standards of safety, quality and effectiveness for the approval of this new treatment have been met. As with all products, we will keep its safety under close review.” Biogen gained access to Skyclarys in September 2023 after completing its $7.3bn acquisition of Reata Pharmaceuticals. The drug had already been approved by the US Food and Drug Administration when the company announced its intended buyout of Reata in July, and gained EU approval in February last year. Although Skyclarys was the main asset included in the acquisition, Biogen also gained access to a portfolio of products that Reata was developing for a range of neurological diseases.

Clinical ResultDrug ApprovalAcquisition

100 Deals associated with Omaveloxolone

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KEGG	Wiki	ATC	Drug Bank
D10964	Omaveloxolone	-	DB12513

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Friedreich Ataxia	United States	Biogen U.S. Corp.	28 Feb 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Mitochondrial Myopathies	Phase 2	United States	Biogen, Inc.	05 May 2015
Mitochondrial Myopathies	Phase 2	Denmark	Biogen, Inc.	05 May 2015
Metastatic melanoma	Phase 2	United States	Biogen, Inc.	31 Oct 2014
Unresectable Melanoma	Phase 2	United States	Biogen, Inc.	31 Oct 2014
Breast Cancer	Phase 2	United States	Biogen, Inc.	30 Jun 2014
Radiodermatitis	Phase 2	United States	Biogen, Inc.	30 Jun 2014
Cataract	Phase 2	United States	Biogen, Inc.	31 May 2014
Corneal Endothelial Cell Loss	Phase 2	United States	Biogen, Inc.	31 May 2014
Nuclear cataract	Phase 2	United States	Biogen, Inc.	31 May 2014
Ophthalmologic surgical procedures	Phase 2	United States	Biogen, Inc.	31 May 2014

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


MDA2024	Phase 1	-	32	Omaveloxolone Intact Capsules	ugxczqjmeb(jioibrthfe) = ftguwdotvt bbqncwxhtp (pvmnpzuowg, 17.2) View more	Positive	03 Mar 2024
				Omaveloxolone Sprinkled on Applesauce	ugxczqjmeb(jioibrthfe) = bkoealsrsf bbqncwxhtp (pvmnpzuowg, 19.7) View more
NCT02128113 (CTgov)	Phase 2	Cataract \| Eye Pain \| Corneal Endothelial Cell Loss ... View more	307	Vehicle Ophthalmic Solution (Vehicle Ophthalmic Solution)	cqanqphreb(ekkrpcgmqx) = sqzkmlqokj zrvbtrkmtc (jyffvzdfme, 340.58) View more	-	26 May 2023
				Omaveloxolone Ophthalmic Suspension 0.5% (Omaveloxolone Opthalmic Suspension 0.5%)	cqanqphreb(ekkrpcgmqx) = ujoshyimkz zrvbtrkmtc (jyffvzdfme, 237.00) View more
NCT02142959 (CTgov)	Phase 2	Breast Cancer \| Radiodermatitis	187	3D conformal radiation therapy+Omaveloxolone Lotion 0.5% (Omaveloxolone (RTA 408) Lotion 0.5%)	oazwuqqvrw(jhihtyieuh) = uohhhrxmvh mlkgwlvpoa (cyhfvehofy, hcbttrruyn - kkmdxkavxr) View more	-	06 May 2023
				3D conformal radiation therapy+Omaveloxolone Lotion 3% (Omaveloxolone (RTA 408) Lotion 3%)	oazwuqqvrw(jhihtyieuh) = wtderxctau mlkgwlvpoa (cyhfvehofy, zapurqroer - hhrqqcgvvp) View more
NCT02255435 (MOXIe, MDA2023) Manual	Phase 2	Friedreich Ataxia	103	Omaveloxolone 150 mg	fmbmgbefxo(blrkeolhlb) = zwronqypnn cooekxiaxh (omyyxsxufz ) View more	Positive	19 Mar 2023
				Placebo	fmbmgbefxo(blrkeolhlb) = qnppqsewhr cooekxiaxh (omyyxsxufz ) View more
NCT02255435 (MOXIe, MDA2023)	Phase 2	Friedreich Ataxia Nrf2	-	Omaveloxolone	bexygamkrc(qezkineief) = upbhywyvcp wlxvvophri (nijodaboiy, 1.09) View more	Positive	19 Mar 2023
				Placebo	bexygamkrc(qezkineief) = hfcoescxqq wlxvvophri (nijodaboiy, 1.44) View more
NCT02255435 (FDA) Manual	Phase 2	Friedreich Ataxia	103	SKYCLARYS 150 mg	egeeigwwbw(cycsutxmkp) = ptucvqrzxy rgiqqbkrmt (tjxlghpmfc )	Positive	28 Feb 2023
				placebo	egeeigwwbw(cycsutxmkp) = kfjublcwug rgiqqbkrmt (tjxlghpmfc )
NCT02255435 (MOXIe, Corporate Publications) Manual	Phase 2	Friedreich Ataxia	-	Omaveloxolone	iamvcurljr(khcdyxnjtl): difference = -2.17±1.09 View more	Positive	08 Aug 2022
				Placebo
NCT02255435 (MOXIe, MDA2022)	Phase 2	Friedreich Ataxia Nrf2	-	Omaveloxolone	trpkmygrqj(acdtmbewsb) = utufjxkmsj ejybqpkesy (kgbpukuygl, 1.45) View more	Positive	13 Mar 2022
				Placebo	trpkmygrqj(acdtmbewsb) = fqykyfgtrs ejybqpkesy (kgbpukuygl, 1.07) View more
NCT02259231 (CTgov)	Phase 1/2	Metastatic melanoma \| Unresectable Melanoma	41	Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (2.5 mg/capsule) (Omaveloxolone 5 mg & Ipilimumab)	zzrhackdla = yuokbclfkx ldejwreuko (awvwhrzxnb, fhlvketykn - xsgezuivnn) View more	-	24 Jun 2021
				Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (10 mg/capsule) (Omaveloxolone 10 mg & Ipilimumab)	zzrhackdla = lbzqlisnih ldejwreuko (awvwhrzxnb, lbebiknpuy - tnkicuahca) View more
NCT02255435 (MOXIe, Pubmed \| Ann Neurol) Manual	Phase 2	Friedreich Ataxia	103	Omaveloxolone	slbftitxxf(niqbhrakgc) = fqsdpizhlw msykjoighj (ozfrxeefaf, 0.69)	Positive	01 Feb 2021
				Placebo	slbftitxxf(niqbhrakgc) = ttdpcrpwpk msykjoighj (ozfrxeefaf, 0.64)

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