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

/ Not yet 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.

NCT06953583

/ Not yet 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 Date03 Jul 2025

Sponsor / Collaborator

Biogen, Inc.

NCT07013292

/ Not yet recruitingNot ApplicableIIT

Retrospective Bicentric Study in a Cohort of French Friedreich's Ataxia Patients on the 6-month Efficacy of Omaveloxolone Treatment on Dysphagia.

Friedreich's ataxia (FA) is a rare, inherited neurodegenerative disease that typically begins in children and young people. It primarily affects the spinal cord, peripheral nerves and cerebellum of the brain. Clinical manifestations include progressive gait and limb ataxia, auditory and optic neuropathy, cardiomyopathy, scoliosis, dysarthria, and dysphagia. In advanced stages, individuals may become wheelchair-dependent, leading to a severe loss of autonomy and reduced life expectancy. To date, there are no effective treatments known to reverse or halt disease progression. Heart disease remains the leading cause of death in individuals with FA. In January 2024, Omaveloxolone was approved for early access in France to treat FA in patients aged 16 years and older. Dysphagia is a central manifestation in FA, and may lead to severe complications such as malnutrition, dehydration, and aspiration-related pneumonia, as well as reduced self-esteem and social isolation. Despite its clinical relevance, dysphagia remains underexplored in clinical trials, including in major Omaveloxolone studies where no specific tool for measuring dysphagia has been incorporated. This study aimed to comprehensively evaluate the effect of Omaveloxolone on dysphagia after six months of treatment, in a cohort of French patients with Friedreich's ataxia who benefited from early access to treatment between February 2024 and May 2025. The severity of dysphagia will be assessed using the Sydney Swallow Questionnaire (SSQ), completed by patients at baseline and after six months of Omaveloxolone treatment.

Start Date01 Jun 2025

Sponsor / Collaborator

Centre Hospitalier Universitaire de Nice

100 Clinical Results associated with Omaveloxolone

100 Translational Medicine associated with Omaveloxolone

100 Patents (Medical) associated with Omaveloxolone

126

Literatures (Medical) associated with Omaveloxolone

01 Jun 2025·MOLECULAR BIOTECHNOLOGY

Omaveloxolone Prevents Polystyrene Microplastic-Induced Ovarian Granulosa Cell Apoptosis via the Keap1/Nrf2/HO-1 Pathway in Rats

Article

Author: Tan, Shu ; Wang, Yan ; Li, Bing ; Yu, Xi

Microplastics (MPs) are persistent environmental pollutants that enter the circulatory system and subsequently reduce sperm quantity and quality. However, the influence of polystyrene MPs (PS-MPs) on the ovary and relevant mechanisms remain elusive. Herein, we aimed to examine the impact of PS-MPs on oxidative disorders in ovarian tissues and elucidate the underlying mechanisms. Healthy female rats were treated with different concentrations of 0.5 µm PS-MPs (diluted in deionized H₂O) for 90 days. Upon examination of hematoxylin-eosin-stained ovarian tissue sections, the number of growing follicles was reduced in PS-MP-treated rats when compared with that in control rats. Enzyme-linked immunosorbent assays revealed that PS-MP exposure markedly reduced anti-Müllerian hormone (AMH) levels. Treatment with PS-MPs downregulated superoxide dismutase, glutathione, and catalase activities in ovarian tissues while upregulating malondialdehyde levels. Furthermore, exposure to PS-MP blocked the Keap1/Nrf2/HO-1 signal transduction pathway. PS-MPs also triggered apoptosis in the ovarian tissue, as evidenced by increased TUNEL staining and expression levels of cleaved caspase-9, Bax, and Bcl-2. To reactivate the Keap1/Nrf2/HO-1 pathway, rats were co-administered PS-MPs and omaveloxolone (Oma), an Nrf2 activator, for 1 week. We found that Oma could counteract the PS-MP-mediated effects on oxidative disorder, apoptosis, AMH production, and follicle number in rat ovarian tissues. To develop an in vitro model, granulosa cells (GCs) were treated with 10 μM H₂O₂ for 12 h to induce oxidative stress. H₂O₂-stimulated GCs exhibited attenuated cell growth and upregulated apoptosis and oxidative stress. Oma administration could ameliorate the H₂O₂-induced effects in terms of regulating cell viability, apoptosis, and oxidative stress in GCs. In summary, PS-MPs could induce apoptosis and oxidative stress via the Keap1/Nrf2/HO-1 signaling pathway in both rats and GCs.

01 Jun 2025·JOURNAL OF CLINICAL PHARMACOLOGY

Clinical Assessment of the Drug–Drug Interaction Potential of Omaveloxolone in Healthy Adult Participants

Article

Author: Hynes, Scott ; Zahir, Hamim ; Valentine, Michelle ; Wu, Lucy ; Murai, Masako

Abstract:

Omaveloxolone is approved in the United States and the European Union for the treatment of patients with Friedreich ataxia aged ≥16 years. It is mainly metabolized by cytochrome P450 (CYP) 3A4 in vitro. Two drug–drug interaction studies (NCT04008186 and NCT05909644) were performed to evaluate (1) the effect of drug‐metabolizing enzymes (DMEs) and drug transporter (DT) modulators on the pharmacokinetics of omaveloxolone and (2) the effect of omaveloxolone on the pharmacokinetics of DME and DT substrates. Additionally, the safety of coadministering these drugs with omaveloxolone was assessed. Coadministration of the strong CYP3A4 inhibitor itraconazole significantly increased omaveloxolone maximum plasma concentration (Cmax) and area under the plasma concentration–time curve from time 0 extrapolated to infinity (AUC0‐∞) by approximately 3‐ and 4‐fold, respectively. Conversely, coadministration with the moderate CYP3A4 inducer efavirenz decreased Cmax and AUC0‐∞ of omaveloxolone by 38.0% and 48.5%, respectively. Omaveloxolone exposure was also increased following coadministration with verapamil, a moderate CYP3A4 and P‐glycoprotein (P‐gp) inhibitor, but it was unaffected by the strong CYP2C8 inhibitor gemfibrozil. Coadministration of omaveloxolone reduced systemic exposure of the substrates of CYP3A4, CYP2C8, breast cancer resistance protein, and organic anion transporting polypeptide 1B1 but had no effect on those of P‐gp and organic cation transporter 1. Omaveloxolone was well tolerated when administered alone and in combination with the DME and DT modulators or substrates. These findings support concomitant medication precautions and dosing recommendations for omaveloxolone when coadministered with a moderate or strong CYP3A4 inhibitor or inducer, as well as the substrates of certain CYP450 enzymes or transporters.

01 Jun 2025·JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Omaveloxolone Suppresses Cell Growth and Causes Cell Cycle Arrest by Downregulating CDC20 Expression in Glioblastoma Cells Both In Vitro and In Vivo

Article

Author: Lieu, Ann‐Shung ; Tsai, Tai‐Hsin ; Hsu, Yi‐Chiang ; Lee, Kuan‐Ting ; Lin, Chih‐Lung

ABSTRACT:

Omaveloxolone is a synthetic oleanane triterpene with considerable antitumor activity. It induces human glioblastoma (GBM) cell death in vitro and in vivo, but the underlying mechanism remains to be determined. In this study, GBM cell lines (GBM8401 and U‐87 MG cells) were exposed to different concentrations of omaveloxolone (0, 600, 800 and 1000 nM). A cell viability assay was conducted using the PrestoBlue Cell Viability Reagent. Three‐dimensional microscopy revealed changes in cell morphology. Cell cycle, apoptosis and mitochondrial membrane potential were tested using flow cytometry. The expression levels of cell cycle‐related proteins and genes were determined through Western blotting and next‐generation sequencing, respectively. The results indicated that omaveloxolone had significant selective cytotoxicity against human GBM cells and suppressed the migration and invasion of these cancer cells. It also caused cell cycle arrest through the downregulation of cell cycle‐related genes, including cell division cycle 20 homologue (CDC20), as revealed by next‐generation sequencing. In a xenograft tumour model, omaveloxolone decreased tumour volume and CDC20 expression. Taken together, these findings suggest that omaveloxolone is a potential drug candidate for GBM treatment by promoting GBM cell death through the downregulation of CDC20 expression.

News (Medical) associated with Omaveloxolone

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

24 Apr 2025

·www.pharmaceutical-technology.com

Biogen’s Friedreich’s ataxia treatment authorised in UK

Biogen’s therapy works by activating the nuclear factor erythroid 2-related factor 2 pathway. Credit: JHVEPhoto / Shutterstock. Biogen Netherlands has received marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its Skyclarys (omaveloxolone) to treat Friedreich’s ataxia in individuals aged 16 years and above. This approval was granted through a national procedure. Friedreich’s ataxia is a widespread hereditary ataxia type, affecting a minimum of one in every 50,000 individuals. It is characterised by progressive issues with balance and movement. A clinical trial that involved 103 subjects demonstrated that individuals with Friedreich’s ataxia, aged between 16 and 40 years, showed neurological improvement when treated with the therapy versus a placebo. After 48 weeks, those on omaveloxolone experienced a reduction in physical impairment. The oral capsule works by activating the nuclear factor erythroid 2-related factor 2 (Nrf2) pathway. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence This protein is typically found at decreased levels in those with Friedreich’s ataxia and is crucial in aiding cellular response to oxidative stress. UK MHRA Healthcare Quality and Access interim executive director Julian Beach said: “Patient safety is our top priority. I am pleased to confirm the first UK approval for the treatment of Friedreich’s ataxia, omaveloxolone. “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.” Skyclarys has been indicated for treating this condition in the adult and adolescent population aged 16 years and above, not only in the UK but also in the European Union, Canada, and the US. The US Food and Drug Administration (FDA) has granted the fast track, orphan drug, and rare paediatric disease designations to the therapy, which also gained orphan drug designation from the European Commission for treating Friedreich’s ataxia. In Canada, Skyclarys was authorised for marketing under the Health Canada Priority Review process.

Orphan DrugDrug ApprovalFast TrackClinical ResultPriority Review

100 Deals associated with Omaveloxolone

External Link

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

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


MDA2024	Phase 1	-	32	Omaveloxolone Intact Capsules	zczcfhkljl(kpkcgrccnm) = grbspnuprb hjcruzbovw (onvrawoetm, 17.2) View more	Positive	03 Mar 2024
				Omaveloxolone Sprinkled on Applesauce	zczcfhkljl(kpkcgrccnm) = vxmbindjrg hjcruzbovw (onvrawoetm, 19.7) View more
NCT02128113 (CTgov)	Phase 2	Cataract \| Eye Pain \| Corneal Endothelial Cell Loss ... View more	307	Vehicle Ophthalmic Solution (Vehicle Ophthalmic Solution)	hacnzerwkg(irpikavcni) = mffkuwrqgu trittdqkys (fhbztrtkst, 340.58) View more	-	26 May 2023
				Omaveloxolone Ophthalmic Suspension 0.5% (Omaveloxolone Opthalmic Suspension 0.5%)	hacnzerwkg(irpikavcni) = woznmhubwh trittdqkys (fhbztrtkst, 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%)	qaxtfrlsbj(xfuzozlbru) = yzdqgpevwg aayjnhmhkp (rzxyegnzsc, hrfxreuhvl - fnkloghuyv) View more	-	06 May 2023
				3D conformal radiation therapy+Omaveloxolone Lotion 3% (Omaveloxolone (RTA 408) Lotion 3%)	qaxtfrlsbj(xfuzozlbru) = nwbpvjamkb aayjnhmhkp (rzxyegnzsc, nlxyhamotv - vqckgvysdb) View more
NCT02255435 (MOXIe, MDA2023) Manual	Phase 2	Friedreich Ataxia	103	Omaveloxolone 150 mg	uctkufqtgh(bfkreaagln) = jtrrdjasxa wikujgnccz (mgjtwazslf ) View more	Positive	19 Mar 2023
				Placebo	uctkufqtgh(bfkreaagln) = xqmefbztyy wikujgnccz (mgjtwazslf ) View more
NCT02255435 (MOXIe, MDA2023)	Phase 2	Friedreich Ataxia Nrf2	-	Omaveloxolone	ijlcqutwei(gmdmyayaxr) = obncxgfqtm wmlituqghv (zgkjkhztir, 1.09) View more	Positive	19 Mar 2023
				Placebo	ijlcqutwei(gmdmyayaxr) = hkipknrqtz wmlituqghv (zgkjkhztir, 1.44) View more
NCT02255435 (FDA) Manual	Phase 2	Friedreich Ataxia	103	SKYCLARYS 150 mg	dzupiftpsj(ruufgtmeav) = cbcajnjnik nxrtguoanp (wvjdxpjsqg )	Positive	28 Feb 2023
				placebo	dzupiftpsj(ruufgtmeav) = gxftxnwwwx nxrtguoanp (wvjdxpjsqg )
NCT02255435 (MOXIe, Corporate Publications) Manual	Phase 2	Friedreich Ataxia	-	Omaveloxolone	qtproizdmt(oqmwqexxya): difference = -2.17±1.09 View more	Positive	08 Aug 2022
				Placebo
NCT02255435 (MOXIe, MDA2022)	Phase 2	Friedreich Ataxia Nrf2	-	Omaveloxolone	eqkbmxepgu(ykqqjnqoau) = jrkzgmmocb alhzjnsbnw (yblkxqpmpw, 1.45) View more	Positive	13 Mar 2022
				Placebo	eqkbmxepgu(ykqqjnqoau) = qpimgihmat alhzjnsbnw (yblkxqpmpw, 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)	ksbepbklfn = cfybxifwgi strwcwyvnp (znvtwaopml, hcsbjhumwq - movlkmqnrt) View more	-	24 Jun 2021
				Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (10 mg/capsule) (Omaveloxolone 10 mg & Ipilimumab)	ksbepbklfn = fwbhgbdzdw strwcwyvnp (znvtwaopml, efpwvooccc - nlrerksmrj) View more
NCT02255435 (MOXIe, Pubmed \| Ann Neurol) Manual	Phase 2	Friedreich Ataxia	103	Omaveloxolone	bqfyzvrudv(pddmselbtf) = nntqdjilwu jrqlxuacqc (ewclthddec, 0.69)	Positive	01 Feb 2021
				Placebo	bqfyzvrudv(pddmselbtf) = vttjzmbqxh jrqlxuacqc (ewclthddec, 0.64)

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