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.

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.

NCT06612879

/ Completed临床1期

An Open-Label, Single Dose Study to Assess the Breast Milk and Plasma Pharmacokinetics of Omaveloxolone (BIIB141) in Healthy Lactating Women

In this study, researchers will learn how BIIB141, also known as omaveloxolone or SKYCLARYS®, moves through the body. This is a drug available for doctors to prescribe for patients with Friedrich's Ataxia. But, this drug has not yet been tested in women who have recently given birth and are breastfeeding or pumping milk for their babies. So, researchers do not know how much of the drug could be passed on to babies through the breastmilk of mothers who may take BIIB141.
The main objective of this study is to learn how a single dose of BIIB141 is processed in both the breastmilk and in the blood of healthy women who are breastfeeding.
The main question researchers want to answer in this study is:
* How does BIIB141 move from the blood into the breastmilk?
Researchers will also learn more about:
* How BIIB141 moves through the blood
* What dose of BIIB141 a baby may get from the mother's breastmilk
* Any medical problems the participants have 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 take a single dose of BIIB141 as a tablet by mouth on Day 1.
* Participants will remain at their study research center for 6 days. During this time, the participants will be provided with an electric breast pump. This is so that the researchers can collect breastmilk samples before and after the participants take BIIB141. The researchers will also collect blood samples.
* After leaving the study research center, the participants will return every 2 days for the next 10 days for more tests and checkups.
* Finally, there will be a follow-up with a "lactation consultant" up to 30 days after each participant's last study visit. This is someone who can help participants with breastfeeding or pumping.
* Each participant will be in the study for up to 2.5 months.

开始日期2024-10-18

申办/合作机构

Biogen, Inc.

100 项与 Omaveloxolone 相关的临床结果

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100 项与 Omaveloxolone 相关的转化医学

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100 项与 Omaveloxolone 相关的专利（医药）

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120

项与 Omaveloxolone 相关的文献（医药）

2025-04-01·Redox Biology

Nrf2 activators for the treatment of rare iron overload diseases: From bench to bedside

Review

作者: Dong, Yimin ; Xiao, Jun ; Ding, Weizhong ; Zheng, Meng ; Li, Feng ; Guan, Hanfeng

Iron overload and related oxidative damage are seen in many rare diseases, due to mutation of iron homeostasis-related genes. As a core regulator on cellular antioxidant reaction, Nrf2 can also decrease systemic and cellular iron levels by regulating iron-related genes and pathways, making Nrf2 activators very good candidates for the treatment of iron overload disorders. Successful examples include the clinical use of omaveloxolone for Friedreich's Ataxia and dimethyl fumarate for relapsing-remitting multiple sclerosis. Despite these uses, the therapeutic potentials of Nrf2 activators for iron overload disorders may be overlooked in clinical practice. Therefore, this study talks about the potential use, possible mechanisms, and precautions of Nrf2 activators in treating rare iron overload diseases. In addition, a combination therapy with Nrf2 activators and iron chelators is proposed for clinical reference, aiming to facilitate the clinical use of Nrf2 activators for more iron overload disorders.

2025-04-01·Nature Reviews Neurology

A global perspective on research advances and future challenges in Friedreich ataxia

Review

作者: Rai, Myriam ; Delatycki, Martin B ; Indelicato, Elisabetta ; Boesch, Sylvia ; Farmer, Jennifer ; França, Marcondes C ; Perlman, Susan

Friedreich ataxia (FRDA) is a rare multisystem, life-limiting disease and is the most common early-onset inherited ataxia in populations of European, Arab and Indian descent. In recent years, substantial progress has been made in dissecting the pathogenesis and natural history of FRDA, and several clinical trials have been initiated. A particularly notable recent achievement was the approval of the nuclear factor erythroid 2-related factor 2 activator omaveloxolone as the first disease-specific therapy for FRDA. In light of these developments, we review milestones in FRDA translational and clinical research over the past 10 years, as well as the various therapeutic strategies currently in the pipeline. We also consider the lessons that have been learned from failed trials and other setbacks. We conclude by presenting a global roadmap for future research, as outlined by the recently established Friedreich's Ataxia Global Clinical Consortium, which covers North and South America, Europe, India, Australia and New Zealand.

2025-03-01·Disease Models & Mechanisms

Robust behavioral assessment of the inducible Friedreich's ataxia mouse does not show improvement with NRF2 induction

Article

作者: Dedkova, Elena N. ; Salinas, Lili ; Montgomery, Claire B. ; Figueroa, Francisco ; Adcock, Lauren E. ; Chang, Tiffany ; Cox, Garrett P. ; Cortopassi, Gino

ABSTRACT:

Friedreich's ataxia, a recessive disorder caused by a mutation in the frataxin (FXN) gene, has few mouse models that demonstrate a progressive behavioral decline paralleling that of patients. A mouse model of systemic frataxin deficiency, the FXNKD, was recently developed using a doxycycline-inducible method; it is thought to mimic the patient phenotype seen when frataxin levels are decreased, but it has not been determined whether it is reliable for assessment of therapeutics. FXNKD mice underwent testing for 12 weeks alongside littermates, undergoing tests of motor function, gait and sensation. Additionally, a subset underwent treatment with omaveloxolone or dimethyl fumarate, both NRF2 inducers. We identified multiple techniques that sensitively detect decline in the mice, including open field, gait analysis and Von Frey tests. Furthermore, we developed a novel Salinas–Montgomery ataxia scale, which allows for more comprehensive assessment than a four-part cerebellar ataxia scale. Despite validating multiple sensitive techniques, we did not see any benefits of NRF2-inducing therapies in any tests. This was exacerbated by the discovery of a sexual dimorphism in FXNKD mice, in which males show more significant decline and better responsiveness to NRF2-inducing therapeutics.

136

项与 Omaveloxolone 相关的新闻（医药）

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

2025-04-17

·蒲公英Ouryao

FDA审评：Omaveloxolone 胶囊溶出高变异原因分析（2）

NDA序号216718申请人Reata Pharmaceuticals 产品名SKYCLARIS (拟定专有名) omaveloxolone 胶囊剂型胶囊规格50mg使用途径口服日最大剂量150mgRx/OTC 原研适应症治疗16岁及以上成人和青少年的Friedreich’s 共济失调症。制剂描述不透明的硬HPMC胶囊，带有浅绿色胶囊壳和蓝色盖子，胶囊壳印有“RTA 408”，胶囊盖白色墨水印有“50”。储存条件20°C - 25°C 溶出变异批内变异如表11所示，所有批次在早期取样时间点（不超过20min）都观察到高变异性。在12粒/批上得到数据的所有批次中，10分钟时间点的%RSD范围为54.4至255.9，15分钟时间点的%RSD范围为22.9至102.8，20分钟%RSD范围为3.4至43.4（包括药物释放非常低的胶囊）。表11: 3期临床注册批次（Omaveloxolone胶囊，50mg）的变异性对比由于批内变异性高，FDA要求企业提供理由，并提供足够支持性数据，证明拟定的溶出方法适用于该制剂。企业在回复FDA的问询时，企业调查了变异性的根本原因，并得出结论，早期取样时间点的较高变异性是由于HPMC胶囊壳在解体/破裂时间上的变异性。企业比较了HPMC和明胶胶囊的破裂时间，发现HPMC胶囊通常有延迟破裂。为了进一步支持omaveloxolone胶囊溶出延迟变异性原因与HPMC胶囊壳的破裂相关，企业比较了带胶囊壳和不带胶囊壳的三批样品溶出曲线特征。如图6所示，不带胶囊壳的内容物溶出时，omaveloxolone 溶出迅速，与完整带有胶囊的样品相比，变异性要低得多，这表明胶囊壳是单个胶囊结果在早期时间点变异性的原因，如宽变异性记录，特别是在10分钟和15分钟。图6: Omaveloxolone 胶囊，50mg，批3173957R，完整带胶囊壳与不带胶囊壳的样品溶出尽管观察到了批次内高变异性，但企业认为这种变异性不太可能 omaveloxolone胶囊的体内药代动力学产生任何影响，因为它是一种BCS IV类化合物，其吸收不受胃排空的显著影响。FDA审评意见：FDA同意企业观点，即观察到的高变异性来自HPMC胶囊的可变的破裂时间。FDA认同，与明胶胶囊相比，HPMC胶囊往往有延迟破裂。然而，企业对HPMC胶囊具有如此高溶解变异性的释释最初没有令FDA满意。可能是HPMC胶囊均一性不好，因此在崩裂时造成了高变异性。从生物药剂学角度看，这可能是一个质量问题。FDA在审评周期内向制剂的审评团队传达了这一担忧。如表12所示，PK参数的变异性超过35%。食物对Cmax和Tmax向左移动（从1小时到5小时）有显著影响，这表明食物通过增加其渗透性显著促进了omaveloxolone的吸收。食物效应研究结果证实，omaveloxolone的渗透性低。由于数据不足，并且该制剂缺乏既定的IVIVC（体外体内相关性），观察到的高批次间溶出变异性对omaveloxolone的PK参数潜在影响无法定量预测。然而，omaveloxolone是一种BCS IV类药物，溶解度低，渗透率低。溶出不是口服吸收速率的限制步骤。考虑到所有制剂粒数在45分钟内实现了接近完全的药物释放，Tmax约为1小时，以及原料药的BCS分类，FDA认为，批次内溶出高变异性不太可能对omaveloxolone的体内药代动力学产生任何显著影响。因此，FDA从生物药剂学角度认为，高溶出变异性不是一个制剂是否批准的影响因素。拟定制剂的临床有效性可接受结果将支持制剂的批准。表12: 血浆omaveloxolone药代动力学参数和药代动力学人群小结（研究408-C-170）批间变异性批间也观察到早期取样时间点的高变异性。特别是，三个注册批次（批次3173381R、3173382R、3173383R）的溶出曲线与四批临床3期样品（批次160033、170103、170114和3173957R）的溶出曲线明显不同，如图7所示。图7: omaveloxolone胶囊，50毫克，注册和临床3期批次溶出放行结果（n=6）图8: omaveloxolone胶囊，50毫克，注册和临床3期批次溶出放行结果（n=12）由于原始提交的注册批次和临床批次取样时间点不同，因此不宜比较其溶出曲线的相似性。根据要求，企业对上述批次重复测试了溶出，并生成具有相同取样时间点的数据，如图8所示。计算出的相似性因子（f2 <50）表明，注册批次的溶出速率明显低于临床批次。由于注册和临床批次之间的上述显著差异和溶出曲线特点，企业被要求提供理由，包括PBBM模型的任何信息，或体内数据信息，确认溶出曲线的显著差异是否会导致体内有效性差异。在IR的回复中，企业表示即使这些批次有不同的溶出曲线，三批胶囊内容物的溶出是相似的，这表明omaveloxolone通常在前10分钟内释放80-90%（图9）。为评估溶出变异性对体内有效性的影响，企业评估了omaveloxolone的药代动力学特征。由于药物吸收速度缓慢，并在11小时（范围为3至24小时）达到最大浓度（Tmax），企业总结溶出曲线在早期时间点的变异性预计不会对omaveloxolone的Tmax产生影响。因此，企业认为溶出曲线的早期时间点（10-20分钟）的变异性将对omaveloxolone的总生物利用度有很小或没有影响。而且，企业正在与（b）（4）建立合作，在禁食条件下研发其体外/体内相关性（IVIVR），并通过传统或建模方法（PBBM）建立安全空间。值得注意的是，企业还没有提供后续信息。图9:批次160033, 3173381R, and 3173857R胶囊内容物的溶出曲线对比FDA进一步查看了数据，发现（b）（4）中生产的胶囊批次药物释放速度往往更快，而（b）（4）中生产的胶囊批次药物释放速度较慢（表10）。这一发现进一步证实，从图9中类似的溶出特点和胶囊内容物的微小变异性来看，溶出变异性来自胶囊破裂时间的变异性，因为这些HPMC胶囊在不同生产地点之间不均匀。表13: 不同生产场地的胶囊壳应用于临床和注册批次信息总结值得注意的是，评估体外溶出如何影响omaveloxolone体内PK的信息有限。只有一批（批次3169638R）用于PK研究。此外，尚未为该制剂建立IVIVC，无法确认溶出较慢的注册批次会导致比临床批次更低的生物利用度。尽管来自三个不同批次胶囊内容物表现出相似的溶出特点（图9），但不能排除由HPMC胶囊引起的不同溶出特点对omaveloxolone的体内有效性产生影响的可能性。在回复FDA的问询中，企业提到他们正在使用PBBM建模和模拟来解决这个问题。关键临床研究中使用的超过4批次胶囊表现出不同的溶出特点，其中少数明显不同。FDA审评员向企业医学官传达了高溶出变异性问题并询问了是否会由于批次内和批次间变异性高而观察到显著不同有效性和安全性特征，以及是否可以进行任何基于批次的子组分析。企业医学官解释到，观察到了可变疗效终点（mFARS）。然而无法得出结论，疗效或安全终点的变异性是否可以归因于体外溶出变异性。FDA与企业进行了会议讨论，结论是根据目前有限的体内数据，很难预测观察到的高批次内和批次间变异性如何影响拟定制剂的PK。尽管这仍是FDA审评员的担忧，但临床结果决定其可得到批准。c）溶出接受标准企业拟定溶出接受标准：Q=(b)(4)在（b)(4)min。FDA审评意见：表10显示，除批次3165601R外，所有临床/注册批次在45分钟内平均溶出至少为87%，这表明拟定的溶出接受标准为(b)(4)，如下：Q= (b)(4) 在45 min。溶出方法区分力研究表明其支持监管决定，设定溶出标准Q= (b)(4) 在45 min可以用于拒绝某类批次（见图5）。如企业对FDA的问询回复所示，企业接受了FDA的溶出接受标准建议。处方桥接如表8所示，临床批次3187136R、3173957R、3169638R、3165601R和3164723R在（b）（4）制造。所有其他批次均在（b）（4）中生产。表14 不同场地生产批次的平均溶出数据比较图10 不同场地生产批次的溶出曲线比较表15 在不同场地生产批次之间的相似性FDA审评，表15中计算的相似性系数表明，除了批次3173957R与批次170103外，在不同场地生产的批次并不相似。请注意，上述所有批次都有用于有效性研究。临床数据将为桥接提供进一步的支持。【解读】审评中发现omaveloxolone胶囊相同批次内的溶出，在前15分钟变异非常大，FDA要求企业提供理由。企业调查研究的原因是胶囊壳在溶出前15分钟的破裂速度有快慢，从而导致批内溶出在前15分钟的高变异性。企业也对比了不使用胶囊壳的内容物溶出，批次之间的速率非常快，不存在变异。化合物的PK数据显示，Tmax为1小时，胶囊的完全溶出时间在45分钟，说明溶出的早期变异性并不影响胶囊在体内的表现，批次内溶出高变异性不太可能对omaveloxolone的体内药代动力学产生显著影响。同样的情形也发生在omaveloxolone胶囊批次之间。不同批次溶出在前15分钟存在高变异性，f2因子对比大部小于50%。原因也是不同批次制剂使用的HPMC胶囊导致，同时HPMC胶囊不同批次之间也存在崩解的差异性。但这些早期时间点溶出高变异性并不影响体内的溶解，因为制剂在体人的Tmax 在3-24小时，前15分钟溶解的变异性不足以对体内产生根本性影响。由于批间和批内溶出的高变异，FDA表示了担忧，并与企业医学负责人进行了会议沟通。但omaveloxolone胶囊在临床上表现出的安全性和有效性良好，药学的变异性不影响其最后的批准。FDA最后也没有要求企业进行胶囊壳成分的变更，或者溶出方法的重新开发，说明新药在FDA得到批准，临床数据的表现会起到关键的作用。推荐文章：1. AI智能体发布大家都在用的“智能体小程序”作者精彩原创集锦1、创新药研发中原料药工艺与沟通交流常见难点2、创新药研发中「制剂工」常见难点3、创新药研发中「变更」相关难点4、创新药研发中「质量分析控制」相关难点（1）5、创新药研发中「质量分析控制」相关难点（2）6、创新药研发难点分析与CMC研究策略探讨7、《已上市化学药品药学变更研究技术指导原则（试行）原料药变更的问答》浅析8、FDA：原料药中亚硝胺风险评估的最新观点9、亚硝胺「传奇」：5年来监管和制药界的反思10、【学习笔记】2023年度CDE审评报告11、FDA审评：吡托布鲁替尼的药学变更桥接(完)12、CDE：ICH指南在国内的实施概况13、「解读」FDA审评：repotrectinib胶囊5次CMC会议内容14、FDA：IND阶段CMC关注点15、药品注册检验难点与建议16、最新 FDA 人用药物亚硝胺杂质指南更新的「底层」参考17、不同国家地区注册检验差异对比曾文亮博士近20年药物研发经验，擅长新药研发与注册申报，近5年积累了近十个小分子新药中美IND申报成功和两个1类抗肿瘤新药上市申报成功经验。先后任职于某大型CRO医药研发公司、USP大中华区研发中心和港资原料药厂等，主导过MNC质量研究项目、USP各论更新和药厂QC实验室通过国家GMP检查和WHO审计。受中国医药保健品进出口商会邀请在二十二届世界制药原料药中国展（CPHI）主导策划了“小分子创新药质量研究和注册策略”沙龙并发表主题演讲；同时受专业药学论坛邀请发表了《创新药质量研究要点和案例分析》、《中美新药申报沟通交流常见问题和案例分析》、《亚硝胺杂质的FDA和EMA控制策略》、《分析方法开发，验证和转移的统计学评价》和《FDA483中清洁验证的典型案例分析》等十余次主题演讲。更多精彩内容可关注：▲ 关注文亮频道，探索未知世界

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100 项与 Omaveloxolone 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10964	Omaveloxolone	-	DB12513

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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

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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

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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	襯窪蓋淵壓觸蓋鑰獵遞(廠鬱鹽鹽淵觸網膚積夢) = 鹹壓獵襯簾製獵遞鹹壓齋網範選齋憲構範齋鹽 (繭艱窪蓋獵衊壓顧鏇鬱, 17.2) 更多	积极	2024-03-03
				Omaveloxolone Sprinkled on Applesauce	襯窪蓋淵壓觸蓋鑰獵遞(廠鬱鹽鹽淵觸網膚積夢) = 餘觸範獵艱網繭觸觸顧齋網範選齋憲構範齋鹽 (繭艱窪蓋獵衊壓顧鏇鬱, 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)	淵窪膚鑰簾觸繭餘選窪(醖壓壓繭鑰夢鏇繭憲膚) = 鑰構鹽遞積窪糧選膚膚夢鹹觸構繭醖膚積齋構 (網構繭糧顧醖鬱遞獵鹽, 340.58) 更多	-	2023-05-26
				Omaveloxolone Ophthalmic Suspension 0.5% (Omaveloxolone Opthalmic Suspension 0.5%)	淵窪膚鑰簾觸繭餘選窪(醖壓壓繭鑰夢鏇繭憲膚) = 鬱醖衊餘鬱夢簾獵遞蓋夢鹹觸構繭醖膚積齋構 (網構繭糧顧醖鬱遞獵鹽, 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%)	淵鑰餘積糧憲獵簾壓願(觸窪夢鹽醖築顧範蓋壓) = 範衊築淵艱糧膚願醖觸壓鹹鹹膚鹽鏇糧膚壓製 (鏇顧繭餘鹹觸壓構範糧, 網選鹽築糧壓選醖鏇膚 ~ 選製淵鹹壓壓積襯鹽鑰) 更多	-	2023-05-06
				3D conformal radiation therapy+Omaveloxolone Lotion 3% (Omaveloxolone (RTA 408) Lotion 3%)	淵鑰餘積糧憲獵簾壓願(觸窪夢鹽醖築顧範蓋壓) = 遞願鹹構選築鏇膚築膚壓鹹鹹膚鹽鏇糧膚壓製 (鏇顧繭餘鹹觸壓構範糧, 築鏇齋積窪築觸繭艱簾 ~ 廠夢鬱衊鑰膚觸齋選範) 更多
NCT02255435 (MOXIe, MDA2023) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone 150 mg	製築遞顧夢夢築鬱廠選(觸繭選衊蓋膚構觸窪鹽) = 獵襯鹹襯淵壓糧獵憲簾衊廠築夢鬱衊鬱壓餘糧 (構範艱鹹遞醖鏇鏇壓夢 ) 更多	积极	2023-03-19
				Placebo	製築遞顧夢夢築鬱廠選(觸繭選衊蓋膚構觸窪鹽) = 網遞構壓鹹繭繭艱網廠衊廠築夢鬱衊鬱壓餘糧 (構範艱鹹遞醖鏇鏇壓夢 ) 更多
NCT02255435 (MOXIe, MDA2023)	临床2期	Friedreich共济失调 Nrf2	-	Omaveloxolone	積夢餘醖遞醖鹽製壓顧(觸製鹽顧築鏇廠獵艱範) = 選構範製淵願壓膚鹹糧淵鑰構衊築鏇鬱製衊齋 (齋繭廠積製鬱範觸願壓, 1.09) 更多	积极	2023-03-19
				Placebo	積夢餘醖遞醖鹽製壓顧(觸製鹽顧築鏇廠獵艱範) = 襯醖製醖鏇網製膚夢鹹淵鑰構衊築鏇鬱製衊齋 (齋繭廠積製鬱範觸願壓, 1.44) 更多
NCT02255435 (FDA) 人工标引	临床2期	Friedreich共济失调	103	SKYCLARYS 150 mg	鏇膚鑰構積鹽範糧艱觸(餘廠淵廠艱遞鏇壓艱壓) = 選獵膚繭夢艱網鬱鑰鏇憲製鹹艱選艱齋淵夢蓋 (築膚壓築齋廠製艱製夢 )	积极	2023-02-28
				placebo	鏇膚鑰構積鹽範糧艱觸(餘廠淵廠艱遞鏇壓艱壓) = 繭廠範遞膚觸鬱夢艱憲憲製鹹艱選艱齋淵夢蓋 (築膚壓築齋廠製艱製夢 )
NCT02255435 (MOXIe, Corporate Publications) 人工标引	临床2期	Friedreich共济失调	-	Omaveloxolone	淵窪遞憲夢壓糧膚積蓋(膚鏇遞網餘簾蓋糧壓築): 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	衊願糧觸願醖築觸廠醖(齋鏇製窪夢蓋鏇選鹹淵) = 繭衊遞膚廠襯憲餘鹹壓鹹淵鏇淵廠衊鏇糧鬱鑰 (窪醖構鏇鬱顧齋餘鹹醖, 1.45) 更多	积极	2022-03-13
				Placebo	衊願糧觸願醖築觸廠醖(齋鏇製窪夢蓋鏇選鹹淵) = 襯範範範鑰構製選糧餘鹹淵鏇淵廠衊鏇糧鬱鑰 (窪醖構鏇鬱顧齋餘鹹醖, 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)	網獵鏇範蓋餘網觸構襯 = 醖艱餘廠鏇繭範壓膚廠蓋鹽憲壓艱獵範鏇憲窪 (憲鬱廠艱選糧選選廠範, 夢構繭製夢繭顧鬱醖膚 ~ 構憲鏇夢醖範構壓襯糧) 更多	-	2021-06-24
				Ipilimumab (3 mg/kg)+Omaveloxolone Capsules (10 mg/capsule) (Omaveloxolone 10 mg & Ipilimumab)	網獵鏇範蓋餘網觸構襯 = 壓築鏇繭窪製獵築窪構蓋鹽憲壓艱獵範鏇憲窪 (憲鬱廠艱選糧選選廠範, 齋顧獵範鹽願製艱繭艱 ~ 鹽鹽範顧顧艱顧糧糧積) 更多
NCT02255435 (MOXIe, Pubmed \| Ann Neurol) 人工标引	临床2期	Friedreich共济失调	103	Omaveloxolone	憲鹽積鹹壓觸餘夢範簾(鑰選製廠簾壓淵繭顧餘) = 齋艱遞積鏇鹹鹽獵顧繭範醖觸蓋衊壓壓蓋觸鬱 (選齋衊淵願鏇願鹽遞願, 0.69)	积极	2021-02-01
				Placebo	憲鹽積鹹壓觸餘夢範簾(鑰選製廠簾壓淵繭顧餘) = 窪鑰築獵衊選範淵範獵範醖觸蓋衊壓壓蓋觸鬱 (選齋衊淵願鏇願鹽遞願, 0.64)