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.

开始日期2026-10-26

申办/合作机构

Biogen, Inc.

NCT06953583

/ Not yet recruiting临床3期

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

开始日期2025-07-03

申办/合作机构

Biogen, Inc.

NCT06623890

/ RecruitingN/A

An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia

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 Friedreich'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 be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time.
The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study.
The main questions researchers want to answer in this study are:
* How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
* How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug?
Researchers will also learn more about :
• Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed
This study will be done as follows:
* Participants will be screened to check if they can join the study.
* After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor.
* During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label.
* Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months.
* Each participant will be in the study for up to 5 years.

开始日期2024-12-12

申办/合作机构

Biogen, Inc.

100 项与 Omaveloxolone 相关的临床结果

登录后查看更多信息

100 项与 Omaveloxolone 相关的转化医学

登录后查看更多信息

100 项与 Omaveloxolone 相关的专利（医药）

登录后查看更多信息

124

项与 Omaveloxolone 相关的文献（医药）

2025-06-01·MOLECULAR BIOTECHNOLOGY

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

Article

作者: Tan, Shu ; Li, Bing ; Wang, Yan ; 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.

2025-06-01·Neurology and Therapy

Safety Monitoring of Omaveloxolone in Friedreich Ataxia: Results from One Year of Clinical Treatment

Article

作者: Keita, Medina ; Wells, McKenzie ; Lynch, David R ; Park, Courtney ; Schadt, Kimberly ; Sharma, Sonal ; Profeta, Victoria ; Gunther, Katherine

INTRODUCTION:

Omaveloxolone, the only approved medication for Friedreich ataxia (FRDA), is an NRF2 activator available since July 2023. We examined safety monitoring of omaveloxolone administration over the first 12 months of administration.

METHODS:

We recorded baseline and follow-up serum transaminase, albumin, total bilirubin, cholesterol, and brain natriuretic peptide (BNP) values as well as adverse events over 1 year in patients initiating commercial omaveloxolone therapy.

RESULTS:

Access to omaveloxolone was obtained in 236 of individuals for whom it was prescribed. Side effects were noted in 23.8% of patient with the most common being gastrointestinal upset, headache, and fatigue baseline. Twenty-one patients (8.9%) permanently discontinued the drug during the first year. Over the first year, 56.6% of patients had at least one transaminase value above the upper limit of normal at some point. Elevations largely occurred over the first 3 months of therapy, and after 6 months of dosing, only 8.6% of patients had elevations in transaminases. Elevations were generally < 3 × the upper limit of normal and decreased with temporary pausing of the drug or dose reduction. Few changes were noted in albumin or bilirubin, and such changes did not parallel changes in transaminases, suggesting they are independent events. BNP values were generally unchanged throughout the year, and no systematic changes in blood counts were noted. Cholesterol and low-density lipoprotein (LDL) elevations were mild.

CONCLUSIONS:

Most patients with FRDA eventually had access to omaveloxolone, and it was generally well tolerated. Side effects were modest, and, overall, most patients remained on the drug. Abnormalities in serum liver function tests were limited to transaminases, resolved with dose pausing or reduction, and diminished markedly over time. Thus, the safety features of omaveloxolone after administration largely resemble the favorable features noted during clinical trials.

2025-04-24·Open Life Sciences

RTA-408 attenuates the hepatic ischemia reperfusion injury in mice possibly by activating the Nrf2/HO-1 signaling pathway

Article

作者: Li, Jindong ; Li, Zhiqin ; Quan, Yao ; Zhang, Fang ; Wang, Jun ; Pan, Bo ; Jiang, Su ; Hua, Huilian

Abstract:

RTA-408, also referred to as Omaveloxolone, is a potent activator of nuclear factor erythroid 2-related factor 2 (Nrf2) and has been demonstrated with protective effects against oxidative stress-induced injury. Oxidative stress is closely associated with the pathogenesis of hepatic ischemia reperfusion injury (HIRI). The aim of this study is to elucidate the impact and underlying mechanisms of RTA-408 in the process of HIRI. In the HIRI mice models, we found that RTA-408 improved liver function of HIRI mice and attenuated the HIRI-induced oxidative stress in vivo. Moreover, the neutrophil infiltration in liver tissues of HIRI mice was alleviated by the administration of RTA-408. RTA-408 treatment also rescued the elevated apoptosis in the liver tissues of HIRI mice. Furthermore, we demonstrated that RTA-408 treatment activated the Nrf2/HO-1 signaling pathway in liver tissues of HIRI mice. Furthermore, the HIRI mice models were developed using Nrf2-deficient mice to explore whether the protective effect of RTA-408 on HIRI was achieved through the activation of Nrf2. The results indicated that RTA-408 did not significantly alleviate the liver injury in Nrf2-deficient mice. Collectively, our results suggest that RTA-408 attenuates HIRI by improving liver function, and attenuating oxidative stress damage, apoptosis and inflammatory response possibly via the Nrf2/HO-1 pathway, which may provide a novel treatment strategy for HIRI patients.

137

项与 Omaveloxolone 相关的新闻（医药）

2025-04-29

·蒲公英Ouryao

一周 | 国际法规药事(04.21-04.27)

◆ ◆ ◆ ◆Paul's Insight2025年4月21日-4月27日◆ ◆ ◆ ◆过去一周（4月21日至4月27日），全球药事监管和行业机构齐齐发声，犹如一场精彩的药品政策与科技盛宴。从FDA的合成色素退市行动，到EMA的罕见病新疗法推荐；从EDQM对生物制品检测标准的升级，到MHRA多款创新疗法的批准；再到行业协会PDA和ISPE在指南与实践层面的多重发力，这一连串新闻不仅彰显了各大机构的监管力度，更映射出制药行业在创新、合规与可持续性间的不断平衡。美国FDA从食药安全到创新药物的“双轨”发力1. 合成食用色素“退场”行动4月22日，美国FDA宣布，计划逐步淘汰美国食品供应中的合成色素（Petroleum-Based Synthetic Dyes），为儿童健康撑起防护伞。FDA表示，这一举措，是“让美国再次健康”（Make America Healthy Again）计划的重要里程碑。此前，1月15日赤藓红（FD&C Red No. 3）禁用已拉开了帷幕，后续这一更广泛的色素退市，将持续推动产业寻求天然、低风险的替代方案。图：赤藓红禁用后，FDA再推新规，八大合成食用色素将退场https://www.fda.gov/news-events/press-announcements/hhs-fda-phase-out-petroleum-based-synthetic-dyes-nations-food-supply2. 药品安全敲响警钟：外用非那雄胺安全红线4月22日，FDA对市场上未经批准的外用非那雄胺配药制剂发出警示，提醒医疗机构、药房及消费者：目前无任何局部非那雄胺产品拥有FDA批准的标签和安全、有效性验证，配制药剂可能隐藏严重风险，包括与米诺地尔等混合制剂的混用场景。FDA直指部分处方药店和远程医疗平台的药品安全风险行为，呼吁行业加强自律与审查。图：就非那雄胺外用安全问题，FDA发布脱发常用药警示https://www.fda.gov/drugs/human-drug-compounding/fda-alerts-health-care-providers-compounders-and-consumers-potential-risks-associated-compounded3. 中国创新药的海外“登台”——派安普利单抗获批4月24日，FDA官网放出重磅消息：康方生物自主研发的PD1单抗派安普利单抗（Penpulimabkcqx）获批，用于复发或转移性非角化型鼻咽癌（NPC）治疗。该产品采用IgG1亚型Fc段改造设计，依托突破性疗法认定、孤儿药资格和快速通道三重加速，联袂正大天晴药业集团布局后续商业化。此举不仅为全球鼻咽癌患者带来曙光，也标志着中国创新药在国际舞台上稳步迈进。（点击此处查看相关阅读）图：中国制药行业又一里程碑，派安普利单抗获FDA批准进入美国市场https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma美国PDA发布新闻季刊4月24日，美国注射剂协会（PDA）发布新闻季刊，总结其在过去一个季度的工作，要点如下。图：PDA发布新闻季刊https://www.pda.org/pda-letter-portal/home/full-article/community-news-quarterly-april-20251）2024年PDA出版奖揭晓PDA颁发奖项给Novartis&Ogilvy团队，表彰其用于无菌性检测的创新方法，该论文名为《一种利用固相流式细胞术进行细胞制备、培养基和缓冲液的快速无菌方法》。年度PDA Letter文章奖项授予武田制药的《理解日本品质》。2）区域分会动态：德国、奥地利和瑞士分会（DACH）亮点纷呈DACH分会“目视检测与颗粒挑战”网络研讨会吸引400余名专家，聚焦AI时代的风险基础方法与质量设计。PDA表示，此次网络研讨会超出预期，如此高的参与度凸显了业界对目视检测和颗粒物相关挑战的关注，同时也强化了DACH分会促进制药行业知识交流和最佳实践的使命。欧盟EMA欧盟罕见病与适应症扩展双管齐下1. DMD新疗法——组蛋白去乙酰化酶（HDAC）抑制剂获“有条件上市许可”4月25日，欧洲药品管理局（EMA）发布杜氏肌营养不良症（DMD）创新药物Duvyzat（givinostat）“有条件上市许可”建议。该组蛋白去乙酰化酶（HDAC）抑制剂在120例6岁以上患者的18个月研究中显著延缓台阶攀爬时间，虽次要终点未全部显著，但整体数据朝积极方向发展。获得“有条件上市许可”，这是欧盟的监管机制之一，旨在促进早期上市药物，以满足未满足医疗需求的。EMA要求申请人后续进行一项随机、安慰剂对照研究，并进行长期随访将补足盲点。上市许可人为Italfarmaco S.p.A.，是是一家总部位于意大利米兰的制药跨国公司。图：杜氏肌营养不良症（DMD）创新药物获“有条件上市许可”https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy2. 甲状腺眼病首创疗法Tepezza获积极审评意见4月25日，EMA对甲状腺眼病（TED）首款靶向IGF1受体的单抗Teprotumumab（Tepezza）给出积极评审意见。三项RCT临床显示活动期患者24周内眼球突出度平均减少2.0–2.3mm，临床活动评分大幅改善；慢性期亦有约1.5mm降低。基于肌肉痉挛、脱发等可管理风险，EMA将附加风险最小化措施并转交欧委会审批。申请人为Amgen Europe B.V.，是全球领先的生物技术公司安进在欧洲的子公司。图：甲状腺眼病首创疗法Tepezza获积极审评意见https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease3. CHMP会议要点：新药、扩展、再审与撤回4月22–25日的EMA人药委员会（CHMP）会议，推荐16款新药（包含Vertex囊性纤维化三联疗法、差异化适应症的多款孤用药及九款生物类似药），推动10项适应症扩展，如武田制药的Adcetris多淋巴瘤适应等；同时，礼来的阿尔茨海默药物Kisunla申请再审，Biohaven和辉瑞的两款产品适应失利撤回。整体来看，EMA通过CMA平衡未满足需求与证据要求，也显示出对成熟产品生命周期管理策略的开放态度。图：2025年4月22日至25日人药委员会( CHMP )会议要点https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025欧洲EDQM高通量测序入典，提升生物制品安全4月23日，EDQM欧洲药典委员会（EPC）正式采纳通则“2.6.41病毒外源性因子检测的高通量测序（HTS）”，自2026年4月1日起生效。新章节涵盖非靶向与靶向HTS方法学、基因组/转录组数据分析和验证指南，驱动与ICH Q5A(R2)修订及WHO国际参考组协同，助力生物制品从传统动物实验迈向更高灵敏度的分子检测。图：高通量测序入欧洲药典https://www.edqm.eu/en/-/epc-adopts-cutting-edge-hts-chapter-to-enhance-viral-contaminant-detection-in-biological-products英国MHRA创新疗法齐发1. 4月拜耳新药Acoramidis获批，用于心肌病4月25日，MHRA批准拜耳制药的Acoramidis（Beyonttra），用于治疗因变异型或野生型转甲状腺素蛋白淀粉样变性（ATTR-CM）引起的心肌病（心肌损伤）成年患者。，632例国际多中心研究显示30个月心血管住院率和死亡率显著降低，6分钟步行距离改善。口服每日两次，通过IRP机制加速审评。图：拜耳新药Acoramidis获批，用于心肌病https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy2. 白血病CAR-T疗法Obecabtagene autoleucel获有条件上市批准4月25日，obecabtagene autoleucel (Aucatzyl) 这一CART疗法获条件上市许可，用于治疗复发或难治性B 细胞前体急性淋巴细胞白血病 (ALL) 的成年患者。FELIX研究153例数据显示12个月总生存率81%、完全缓解55%，后续需提供更多数据满足全面上市要求。上市许可人为Autolus Therapeutics，是一家总部位于英国的生物制药公司，该公司于2014 年从伦敦大学学院（UCL）分拆成立。图：白血病CAR T疗法Obecabtagene autoleucel获有条件上市批准https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia3. 百健新药在英获批4月23日，MHRA批准了 omaveloxolone（Skyclarys），这是英国首个针对 16 岁及以上患者的治疗药物，用于治疗一种名为弗里德赖希共济失调的罕见神经退行性运动障碍。弗里德赖希共济失调是最常见的遗传性共济失调（由遗传基因引起）。据估计，每5万人中至少有1人患有此病。弗里德赖希共济失调的症状包括平衡和运动障碍，并会随着时间的推移逐渐恶化。该药物为口服胶囊剂。上市许可人为Biogen Netherlands B.V.，为全球领先的生物技术公司 Biogen Inc.（百健公司）在荷兰的全资子公司。图：百健新药在英获批https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-friedreichs-ataxia-omaveloxolone4. 辉瑞血友病药物获批4月22日，MHRA批准辉瑞药物Marstacimab（Hympavzi），用于预防或减少12岁及以上、体重至少35公斤的血友病A和B患者的出血。该药物是同类药物中第一个通过针对血液凝固过程中的蛋白质来发挥作用的药物。Marstacimab每周一次皮下注射，使用预充式注射器或注射笔。患者或护理人员经过适当培训后即可自行注射药物。图：辉瑞血友病药物获批https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b国际ISPE发布数字化验证良好实践指南国际制药工程协会（ISPE）发布了《数字化验证良好实践指南》（Good Practice Guide: Digital Validation），为制药企业提供从传统验证向数字化框架过渡提供了指导。这本90页的指导文件，旨在帮助制药企业采用数字化验证工具（Digital Validation Tools, DVT），提升数据可靠性、运营效率和监管合规性。该指南由全球专家团队开发，其核心成员来自于武田制药、葛兰素史克等全球药企和行业咨询公司。ISPE表示，指南结合真实案例和结构化方法，适用于从初创到优化现有系统的各类企业。（点击此处查看相关阅读）图：ISPE最新发布《数字化验证良好实践指南》https://ispe.org/publications/guidance-documents/good-practice-guide-digital-validation结语回望过去一周，FDA、EMA、EDQM、MHRA、PDA和ISPE等监管与行业机构的多项决策、指南与更新，彰显监管严谨，又鼓励技术革新。从食品安全到精准疗法，从分子检测到数字化治理，昭示出全球制药行业正朝着更健康、更高效、更可持续的未来协奏。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月6日 | 线上 | 新法规和应用场景下的全球包材注册策略和最佳实践5月8日 | 线上 | 双维度解锁无菌药品生产污染防控策略

孤儿药突破性疗法上市批准医药出海申请上市

2025-04-24

·PMLiVE

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.

临床结果上市批准并购

2025-04-24

·Pharmaceutical Technology

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.

孤儿药上市批准快速通道临床结果优先审批

100 项与 Omaveloxolone 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D10964	Omaveloxolone	-	DB12513

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
Friedreich共济失调	美国	Biogen U.S. Corp.	2023-02-28

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
线粒体肌病	临床2期	美国	Reata Pharmaceuticals, Inc.	2015-05-05
线粒体肌病	临床2期	丹麦	Reata Pharmaceuticals, Inc.	2015-05-05
转移性黑色素瘤	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-10-31
不可切除的黑色素瘤	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-10-31
乳腺癌	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-06-30
放射性皮炎	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-06-30
白内障	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-05-31
角膜内皮细胞损失	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-05-31
眼科外科手术	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-05-31
眼睛疼痛	临床2期	美国	Reata Pharmaceuticals, Inc.	2014-02-28

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


MDA2024	临床1期	-	32	Omaveloxolone Intact Capsules	襯蓋衊簾鬱鑰鹹衊淵網(餘築蓋鏇蓋鹽醖襯窪鹽) = 糧衊餘鹽願觸憲齋鏇鑰簾網糧鹹製遞構鹽鏇衊 (鏇廠願淵淵築廠網鬱衊, 17.2) 更多	积极	2024-03-03
				Omaveloxolone Sprinkled on Applesauce	襯蓋衊簾鬱鑰鹹衊淵網(餘築蓋鏇蓋鹽醖襯窪鹽) = 艱鏇範壓鏇鏇憲遞蓋遞簾網糧鹹製遞構鹽鏇衊 (鏇廠願淵淵築廠網鬱衊, 19.7) 更多
NCT02128113 (CTgov)	临床2期	白内障 \| 眼睛疼痛 \| 眼部炎症 ... 更多	307	Vehicle Ophthalmic Solution (Vehicle Ophthalmic Solution)	淵壓衊遞簾壓顧窪衊淵(蓋製窪醖憲襯構遞積構) = 顧衊築衊獵齋窪範餘廠夢醖廠範齋鏇願廠鬱網 (觸齋齋醖餘憲構膚廠淵, 340.58) 更多	-	2023-05-26
				Omaveloxolone Ophthalmic Suspension 0.5% (Omaveloxolone Opthalmic Suspension 0.5%)	淵壓衊遞簾壓顧窪衊淵(蓋製窪醖憲襯構遞積構) = 繭糧遞願積願膚醖衊鹽夢醖廠範齋鏇願廠鬱網 (觸齋齋醖餘憲構膚廠淵, 237.00) 更多
NCT02142959 (CTgov)	临床2期	乳腺癌 \| 放射性皮炎	187	3D conformal radiation therapy+Omaveloxolone Lotion 0.5% (Omaveloxolone (RTA 408) Lotion 0.5%)	齋鏇衊窪淵鹽範窪網憲(廠鹽廠鹹顧艱鹹積鹽壓) = 餘鬱築膚壓艱鏇簾糧獵鑰衊醖願糧積淵蓋繭願 (淵襯鑰餘壓簾製遞積鹽, 艱鏇鬱鹹製願簾獵鑰網 ~ 遞鹹糧齋鬱簾醖製獵觸) 更多	-	2023-05-06
				3D conformal radiation therapy+Omaveloxolone Lotion 3% (Omaveloxolone (RTA 408) Lotion 3%)	齋鏇衊窪淵鹽範窪網憲(廠鹽廠鹹顧艱鹹積鹽壓) = 壓艱夢遞鬱餘糧簾觸遞鑰衊醖願糧積淵蓋繭願 (淵襯鑰餘壓簾製遞積鹽, 顧築構壓襯廠壓壓觸淵 ~ 憲觸衊鹹廠願願製壓壓) 更多
NCT02255435 (MOXIe, MDA2023) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone 150 mg	膚顧膚鬱齋鬱衊蓋範壓(遞鹽鏇築繭膚願膚選築) = 鑰鬱繭鏇獵襯艱憲醖艱齋憲糧選鑰網構選獵糧 (鹽願構選遞艱襯遞積範 ) 更多	积极	2023-03-19
				Placebo	膚顧膚鬱齋鬱衊蓋範壓(遞鹽鏇築繭膚願膚選築) = 獵鹹齋繭膚遞鹽憲願鑰齋憲糧選鑰網構選獵糧 (鹽願構選遞艱襯遞積範 ) 更多
NCT02255435 (MOXIe, MDA2023)	临床2期	Friedreich共济失调 Nrf2	-	Omaveloxolone	衊鏇醖膚範鬱蓋窪餘鑰(糧網網積憲願觸獵顧繭) = 蓋衊構膚鏇範窪齋餘鹽餘鑰觸獵鑰鏇齋襯獵廠 (鹹製願糧繭範獵願夢獵, 1.09) 更多	积极	2023-03-19
				Placebo	衊鏇醖膚範鬱蓋窪餘鑰(糧網網積憲願觸獵顧繭) = 壓範衊觸憲鏇廠選構製餘鑰觸獵鑰鏇齋襯獵廠 (鹹製願糧繭範獵願夢獵, 1.44) 更多
NCT02255435 (FDA) 人工标引	临床2期	Friedreich共济失调	103	SKYCLARYS 150 mg	鏇鹽築艱簾築選憲醖衊(醖獵蓋窪膚簾艱鑰廠鑰) = 淵鏇繭醖遞範餘艱鏇餘構築夢窪選糧艱簾獵壓 (鑰憲製構選夢衊願願憲 )	积极	2023-02-28
				placebo	鏇鹽築艱簾築選憲醖衊(醖獵蓋窪膚簾艱鑰廠鑰) = 糧鹹鑰製夢廠製積遞壓構築夢窪選糧艱簾獵壓 (鑰憲製構選夢衊願願憲 )
NCT02255435 (MOXIe, Corporate Publications) 人工标引	临床2期	Friedreich共济失调	-	Omaveloxolone	壓選壓築觸遞築願醖齋(願範築糧廠廠夢鹽艱糧): difference = -2.17±1.09 更多	积极	2022-08-08
				Placebo
NCT02255435 (MOXIe, MDA2022)	临床2期	Friedreich共济失调 Nrf2	-	Omaveloxolone	構壓獵積觸夢衊窪鏇鑰(選憲鹹糧願壓製觸餘壓) = 鹽積衊鬱構鹹選壓獵窪鬱糧鬱夢艱鏇顧構襯憲 (願選範襯鬱網鹹鏇鏇網, 1.45) 更多	积极	2022-03-13
				Placebo	構壓獵積觸夢衊窪鏇鑰(選憲鹹糧願壓製觸餘壓) = 壓鑰網觸構膚淵鏇構鏇鬱糧鬱夢艱鏇顧構襯憲 (願選範襯鬱網鹹鏇鏇網, 1.07) 更多
NCT02259231 (CTgov)	临床1/2期	转移性黑色素瘤 \| 不可切除的黑色素瘤	41	Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (2.5 mg/capsule) (Omaveloxolone 5 mg & Ipilimumab)	顧積顧鏇製醖蓋淵壓顧 = 窪鹽醖襯衊膚網觸廠顧窪膚選襯築憲獵廠窪簾 (艱餘鑰鑰構願淵蓋繭鑰, 窪鹹齋醖餘窪餘窪簾繭 ~ 餘鹽製鹽繭襯襯構淵糧) 更多	-	2021-06-24
				Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (10 mg/capsule) (Omaveloxolone 10 mg & Ipilimumab)	顧積顧鏇製醖蓋淵壓顧 = 淵顧積憲觸淵衊蓋憲繭窪膚選襯築憲獵廠窪簾 (艱餘鑰鑰構願淵蓋繭鑰, 鹹範憲顧觸蓋鬱膚鑰夢 ~ 鑰醖鏇觸艱築醖鹹鬱築) 更多
NCT02255435 (MOXIe, Pubmed \| Ann Neurol) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone	繭襯觸廠糧築願積築鹽(餘壓積廠繭鏇顧簾齋網) = 窪襯顧構艱齋遞願艱餘築鑰繭簾餘襯築網壓壓 (夢糧築壓艱製獵鹹醖淵, 0.69)	积极	2021-02-01
				Placebo	繭襯觸廠糧築願積築鹽(餘壓積廠繭鏇顧簾齋網) = 鑰觸糧鬱築選餘築醖醖築鑰繭簾餘襯築網壓壓 (夢糧築壓艱製獵鹹醖淵, 0.64)