Identification, Assessment, and Treatment of Tardive Dyskinesia With Deutetrabenazine in Adults With Intellectual/Developmental Disabilities and Co-occurring Psychiatric and/or Behavioral Disorders

The primary goal of this study is to investigate the efficacy of deutetrabenazine treatment of TD in this previously untreated patient population. Compare movement disorder deutetrabenazine treatment response in persons with IDD to response seen in patients without IDD treated with deutetrabenazine in other treatment settings (per literature review). Compare global deutetrabenazine treatment response with validated instruments.
In addition, we plan to:
* Assess the safety of deutetrabenazine in the treatment of TD in persons with IDD.
* Assess change in Activities of Daily Living (ADLs) in persons with IDD and TD treated with deutetrabenazine, utilizing a validated ADL instrument.
* Assess change in Quality of Life (QOL) in persons with IDD and TD treated with deutetrabenazine, utilizing a validated QOL instrument.
* Assess caregiver burden with a validated caregiver burden instrument.
In this study, 25 participants with IDD and TD will undergo Deutetrabenazine treatment for 24 weeks. The participants will be seen for a total of 5 visits: at baseline, and at follow up visits at 3 weeks, 6 weeks, 12 weeks, and 24 weeks.
This study does not include a comparison group. Therefore, researchers will compare the response of the study participants to deutetrabenazine treatment with those from a previous reported work that resulted in the FDA approval of this medication. This will be an open-label, Phase 4 study.

开始日期2026-05-01

申办/合作机构

University Hospitals Cleveland Medical Center

[+1]

NCT07601516

/ Completed临床4期

Real World Effectiveness and Safety of Deutetrabenazine in Chinese Patients With Huntington's Disease (HD) Chorea

The Primary Objective: To evaluate the real-world effectiveness of deutetrabenazine in adult patients with chorea associated with Huntington's disease in China.
The Secondary Objectives: To evaluate the real-world safety of deutetrabenazine in adult patients with chorea associated with Huntington's disease in China.

开始日期2024-02-02

申办/合作机构

Teva Branded Pharmaceutical Products R&D LLC

CTR20210396

/ Completed临床4期

安泰坦（氘丁苯那嗪）治疗成人迟发性运动障碍有效性及安全性的中国真实世界研究

评估安泰坦（氘丁苯那嗪）治疗中国成人迟发性运动障碍患者的安全性数据

开始日期2023-11-13

申办/合作机构

Anesta AG

[+2]

100 项与氘丁苯那嗪相关的临床结果

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100 项与氘丁苯那嗪相关的转化医学

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100 项与氘丁苯那嗪相关的专利（医药）

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项与氘丁苯那嗪相关的文献（医药）

2026-06-01·EUROPEAN JOURNAL OF PHARMACOLOGY

Beyond motor symptoms: efficacy of pharmacotherapies on motor, functional, and cognitive domains in Huntington's disease - a systematic review

Review

作者: Teixeira, Amanda Gomes ; Wiprich, Melissa Talita ; Colonetti, Tamy ; Endler, Ana Thereza ; Rosa, Maria Inês da ; Bonan, Carla Denise ; Pereira, Wuesllen Dondossola ; Lodetti, Guilherme ; Rico, Eduardo Pacheco ; Pacagnan, Lucas de Oliveira ; Redivo, Henrique

INTRODUCTION:

Huntington's disease (HD) is a progressive neurodegenerative disorder characterized by motor, neuropsychiatric, cognitive, and peripheral alterations impairing functional capacity and quality of life (QoL). Pharmacotherapies, including dopaminergic and glutamatergic drugs, primarily target motor symptoms, while cholinergic modulators address cognitive decline. However, the literature lacks comprehensive reviews evaluating the combination of these three drug classes across a broad spectrum of clinical outcomes, extending beyond the total motor score (TMS).

OBJECTIVE:

This systematic review aimed to assess the efficacy of dopaminergic, glutamatergic, and cholinergic drugs on motor, functional, cognitive decline, and QoL in HD patients.

METHODOLOGY:

Following PRISMA guidelines, searches were performed in MEDLINE, Embase, Scopus, Web of Science, and the Cochrane Library until October 2025. Randomized clinical trials evaluating these three classes of drugs in adults with genetically confirmed HD were included. Primary outcomes were Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS), Total Functional Capacity (TFC), and cognitive performance. Secondary outcomes included the Independence Scale (IS) and QoL. Risk of bias was assessed using the Cochrane RoB 2 tool. Due to substantial heterogeneity, results were synthesized descriptively.

RESULTS:

From 32,746 studies, 15 were included in the final analysis. Findings showed that UHDRS TMS was improved with dopaminergics (tetrabenazine, deutetrabenazine, and pridopidine) and glutamatergic (riluzole) drugs. Only pridopidine treatment exhibited efficacy on UHDRS TFC, subsequently improving UHDRS IS. For QoL, deutetrabenazine treatment proved effective. In the UHDRS cognitive domain, tetrabenazine improved performance in the Stroop Color and Word test, while rivastigmine (cholinergic) improved verbal fluency.

CONCLUSION:

Dopaminergic, glutamatergic, and cholinergic drugs exhibit distinct efficacy profiles across various motor and non-motor UHDRS components. This positions them as therapeutic options for attenuating both the motor and non-motor symptoms in HD.

2026-04-01·PARKINSONISM & RELATED DISORDERS

Safety and efficacy of VMAT2 inhibitors in Huntington Disease: A systematic review

Review

作者: Dashtipour, Khashayar ; Koch, Jessa ; Dehnavi, Ali Zare ; Baghaei, Ava ; Hashempour, Zahra ; Jimenez-Shahed, Joohi ; Stimming, Erin Furr ; Bordelon, Yvette

BACKGROUND:

Chorea affects quality of life and functional independence in most adults with Huntington disease (HD). Vesicular monoamine transporter 2 (VMAT2) inhibitors, tetrabenazine, deutetrabenazine, and valbenazine, are pharmacological agents used to manage HD-related chorea. This systematic review evaluates the safety and efficacy of these agents in individuals with HD.

METHODS:

This review was conducted in accordance with PRISMA guidelines and registered with PROSPERO, CRD42024581306. A comprehensive literature search was performed for studies published up to July 3, 2025. Eligible studies included randomized controlled trials, cohort studies, and case-control studies. Two reviewers independently performed data extraction and risk-of-bias assessment.

RESULTS:

Three randomized trials met eligibility criteria and were included in the final analysis. All three VMAT2 inhibitors significantly reduced Unified Huntington Disease Rating Scale Total Maximal Chorea scores. Clinical Global Impression-Improvement Scale for tetrabenazine and both Patient Global Impression of Change and Clinician Global Impression of Change ratings for deutetrabenazine and valbenazine favored active treatment. Deutetrabenazine modestly improved 36-Item Short Form Health Survey physical functioning and Swallowing Disturbance Questionnaire scores. Tetrabenazine was associated with higher adverse event (AE) rates; whereas valbenazine and deutetrabenazine demonstrated better tolerability.

DISCUSSION:

This systematic review shows VMAT2 inhibitors significantly reduce chorea severity in HD. While tetrabenazine showed the greatest reduction in chorea scores, valbenazine and deutetrabenazine provided broader benefits on functional outcomes and improvement scales. Tetrabenazine had the highest rate of AEs, whereas deutetrabenazine demonstrated favorable tolerability, with fewer complications. Valbenazine demonstrated an intermediate safety profile. These differences should guide individualized treatment decisions in clinical practice.

2026-03-01·Clinical Pharmacology in Drug Development

Pharmacokinetics and Bioequivalence of 2 Deutetrabenazine Tablets in Healthy Chinese Volunteers Under Fasting and Fed Conditions

Article

作者: Li, Xin ; Wei, Liying ; Xu, Dayong ; Wang, Pengkai ; Zhang, Ping ; Xiong, Xiaolian ; Li, Yuan

Abstract:

Deutetrabenazine, a deuterated vesicular monoamine transporter 2 (VMAT2) inhibitor, is approved for the treatment of Huntington's disease (HD)‐related chorea and tardive dyskinesia (TD). However, pharmacokinetics (PK) and bioequivalence (BE) evidence in the Chinese population has been lacking. This single‐center, randomized, open‐label, single‐dose, two‐formulation, four‐period, fully replicated crossover study evaluated the PK, BE, and safety of test and reference deutetrabenazine 12 mg tablets under fasting and fed conditions in healthy Chinese volunteers. A total of 90 subjects were enrolled (40 fasting; 50 fed), and 88 completed the study. Plasma concentrations of deutetrabenazine and its active metabolites d6‐α‐dihydrotetrabenazine (d6‐α‐HTBZ) and d6‐β‐dihydrotetrabenazine (d6‐β‐HTBZ) were quantified using a validated liquid chromatography‐tandem mass spectrometry method. BE was assessed using average bioequivalence (ABE) or reference‐scaled average bioequivalence (RSABE) according to within‐subject variability. Under fasting conditions, RSABE was applied for maximum plasma concentration (C max ) and met equivalence criteria (point estimate within 80%–125%, upper confidence interval bound ≤ 0); under fed conditions, as well as for all remaining PK parameters, ABE criteria were met with the 90% confidence interval within the 80%–125% range. Both formulations were well tolerated, with only mild and transient adverse events and no serious safety findings. These results demonstrate that the test and reference deutetrabenazine tablets are bioequivalent in healthy Chinese adults under both nutritional states.

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项与氘丁苯那嗪相关的新闻（医药）

2026-06-25

·梯瓦医药

安泰坦XR®在华获批，每日一次缓释剂型助力亨廷顿病（HD）有关的舞蹈病及迟发性运动障碍（TD）长期管理

安泰坦XR®（通用名：氘丁苯那嗪缓释片）在华获批，用于治疗成人与亨廷顿病有关的舞蹈病及成人迟发性运动障碍。作为安泰坦®的重要升级剂型，安泰坦XR®为每日一次缓释剂型，有助于简化用药方案，支持患者长期治疗管理。氘丁苯那嗪III期临床试验（ARM-TD研究和AIM-TD研究）均显示，氘丁苯那嗪可显著改善迟发性运动障碍患者异常不自主运动症状；ARM-TD研究中，患者异常不自主运动量表评分较安慰剂显著改善1；AIM-TD研究中，24mg/日和36mg/日剂量组显示显著疗效，且整体耐受性良好2。 6月25日，梯瓦医药宣布安泰坦XR®（AUSTEDO XR®, 通用名：氘丁苯那嗪缓释片）经中国国家药品监督管理局批准，用于治疗成人与亨廷顿病（HD）有关的舞蹈病及成人迟发性运动障碍（TD）。作为已在中国上市的安泰坦®（Austedo®, 通用名: 氘丁苯那嗪片）的重要升级剂型，安泰坦XR®以每日一次缓释剂型进一步简化用药方案，为患者带来更便捷、更平稳的长期管理选择。亨廷顿病有关的舞蹈病及迟发性运动障碍均为需要长期持续控制的慢性疾病3,4 。在临床实践中，用药依从性是影响治疗效果的重要因素之一5,6,7。研究显示，约60%–80%的迟发性运动障碍患者存在口-舌-颊综合征，可能增加进食及服药难度8；而亨廷顿病有关的舞蹈病患者中，约90.5%可能出现吞咽障碍9。对于需长期治疗的患者而言，疾病症状带来的服药困难、给药频次与剂量调整等用药管理要求，往往进一步加重患者及照护者的日常管理负担。安泰坦XR®采用内控释、外速释的双层释放，由速释外层包衣与控释渗透片芯组成；其中，控释片芯应用双室渗透泵技术，可实现药物在体内持续释放。在与常释片疗效等效、安全性相似的同时，安泰坦XR®提供每日一次给药方案，并有助于维持更平稳的血药浓度。基于这一剂型升级，患者可进一步减少服药片数、且无需随餐服用；同时，不同规格组合也支持更灵活的剂量调整，从而有助于简化用药管理、提升治疗依从性，更好地满足患者及照护者的长期管理需求，实现治疗从“症状控制”向“长期规范管理”的临床价值延展。黄迪仁梯瓦医药大中华区总经理 “对需要长期治疗的患者而言，治疗价值不仅在于获得有效药物，也在于建立稳定、便捷的日常管理方式。安泰坦XR®的获批，正是在安泰坦®既有临床价值基础上的进一步延展。我们希望通过每日一次的缓释剂型，进一步减轻患者长期用药管理负担，让优质治疗方案更好地融入患者日常生活。” 氘丁苯那嗪是一种选择性的囊泡单胺转运体2（VMAT-2）口服小分子抑制剂，可通过阻止多巴胺进入突触前神经元的囊泡，调节突触前多巴胺水平，从而帮助控制异常不自主运动10。作为全球首个获批的氘代药物，氘丁苯那嗪基于氘代分子修饰技术，获得包括FDA突破性疗法认定在内的多项创新性认可11。在亨廷顿病相关舞蹈病领域，《中国亨廷顿病诊治指南2023》将氘丁苯那嗪列为治疗舞蹈样动作的一线治疗药物12；在迟发性运动障碍领域，安泰坦®为目前国内唯一获批TD适应症药物13，也是《中国精神分裂症防治指南(第三版)》等国内外临床指南推荐的一线治疗药物14。2026年精神分裂症国际研究协会（SIRS）年会发布的中国首个氘丁苯那嗪多中心IV期真实世界研究显示，接受氘丁苯那嗪治疗后，TD患者异常不自主运动严重程度平均改善达53.8%；其中，≥24mg/日剂量组平均改善达60.1%，且整体耐受性良好。长期以来，梯瓦始终密切关注中国患者在神经系统疾病领域的未尽需求，持续推动全球优质医疗资源在中国的引入与落地。从安泰坦®到安泰坦XR®，梯瓦不断拓展相关疾病领域的用药选择，并将继续提升创新方案可及性，为更多中国患者带来持续、可靠的治疗支持，助力改善患者体验与生活质量。关于安泰坦XR®/氘丁苯那嗪缓释剂型安泰坦XR®（AUSTEDO XR®, 通用名：氘丁苯那嗪缓释片）是安泰坦®的每日一次缓释剂型，属于选择性囊泡单胺转运体2（VMAT-2）口服小分子抑制剂，用于治疗成人与亨廷顿病有关的舞蹈病及成人迟发性运动障碍，并被国内外指南推荐为上述疾病领域的一线治疗选择。作为已在中国上市的安泰坦®的重要升级剂型，安泰坦XR®采用内控释、外速释的双层释放系统，内控层采用双室渗透泵技术，实现每日一次给药，有助于维持更平稳的血药浓度，进一步优化长期用药管理体验。关于梯瓦医药梯瓦医药正在加速转型为一家全球领先的生物医药企业，依托其全球一流的仿制药与生物类似药业务基础。逾120年来，梯瓦对构筑人类健康未来的承诺从未动摇。无论是持续推进在神经科学、免疫学等领域的创新研发，还是在全球范围内提供高质量的复杂仿制药、生物类似药及非处方药产品，梯瓦始终致力于满足患者当下及未来的治疗需求。患者所需，即梯瓦所向。梯瓦全力以赴，共筑健康未来。向上滑动了解更多参考文献： 1.Fernandez HH, Factor SA, Hauser RA, et al. Randomized controlled trial of deutetrabenazine for tardive dyskinesia: The ARM-TD study. Neurology. 2017;88(21):2003–2010 2.Anderson KE, Stamler D, Davis MD, et al. Deutetrabenazine for treatment of involuntary movements in patients with tardive dyskinesia (AIM-TD): a double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Psychiatry. 2017;4(8):595–604 3.Colosimo C, et al. Eur Neuropsychopharmacol. 2026 Mar;104:112740 4.袁俊亮, 等. 中华精神科杂志. 2024;57(2)121-128 5.Caroff SN. et al. Neuropsychiatr Dis Treat. 2019 Apr 4;15:785-794 6.2025 ISPOR-Assessing real-world dosing patterns for VMAT2i, valbenazine and deutetrabenazine, among persistent patients with tardive dyskinesia in a nationwide US claims database 7.Chen S, et al. Neuroepidemiology. 2024;58(1):31-36 8.Hauser RA, et al. CNS Spectr. 2022 Apr;27(2):208-217 9.Kalkers K, et al. J Nutr Health Aging. 2022;26(4):332-338 10.Dorfman BJ, Jimenez-Shahed J. Expert Rev Neurother. 2021 Jan;21(1):9-20. 11.https://ir.tevapharm.com/news-and-events/press-releases/press-release-details/2015/Teva-Announces-Breakthrough-Therapy-Designation-for-SD-809-Granted-by-FDA-for-the-Treatment-of-Tardive-Dyskinesia/default.aspx 12. 中华医学会神经病学分会神经遗传学组. 中国亨廷顿病诊治指南2023[J]. 中华神经科杂志, 2023, 56(8): 848-855. DOI: 10.3760/cma.j.cn113694-20221226-00968. 13.梯瓦医药. (2023). 氘丁苯那嗪片说明书 (修订版) 14.刘登堂,司天梅,况利,等.氘丁苯那嗪治疗迟发性运动障碍临床应用指导建议[J].中国神经精神疾病杂志,2025,51(02):65-71. 审批编号：AUST-CN-01126

2026-06-23

·allsci.com

Teva files Emalex’s ecopipam with US FDA for first approval as Tourette’s therapy

Teva Pharmaceutical Industries (NYSE: TEVA) has submitted a New Drug Application (NDA) to the US FDA for ecopipam, an investigational selective dopamine D1 receptor antagonist, seeking approval for the treatment of pediatric Tourette syndrome. If approved, ecopipam would represent the first new FDA-authorized therapy for pediatric tic disorder in more than a decade and the first agent in clinical use to target the D1 receptor subtype in this indication. The filing arrives weeks after Teva closed its acquisition of Emalex Biosciences, the Chicago-based company that developed ecopipam through Phase III, in a deal valued at up to USD 900 million. The NDA is a standard submission seeking approval for ecopipam in pediatric patients with Tourette syndrome, the company said. Ecopipam has previously received both Orphan Drug Designation and Fast Track Designation from the FDA for this indication. The Orphan Drug Designation reflects the patient population of fewer than 200,000 in the US, and the Fast Track Designation is intended to facilitate a more expedited review process. No PDUFA target action date has yet been disclosed, as FDA acceptance of the filing has not been confirmed. The NDA is supported by Phase III data published in JAMA Neurology in May 2026, which reported results from a randomized, placebo-controlled withdrawal study in pediatric patients. The trial enrolled 216 participants, including 167 children and adolescents, who received open-label ecopipam for 12 weeks. Those who achieved a clinical response were then randomized to continue ecopipam or switch to placebo for a further 12 weeks. The primary efficacy endpoint was time to relapse, measured using the Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS). The study demonstrated a statistically significant benefit for ecopipam over placebo in the pediatric population, with a reported p-value of 0.008. Continued ecopipam was associated with a 50% reduction in relapse risk compared with placebo, the company said. The safety profile was described as generally well tolerated; the most frequently reported adverse events in the ecopipam arm included somnolence (11.1%), anxiety (9.7%), headache (9.7%), insomnia (8.8%), tic (7.9%), and fatigue (6.5%). Ecopipam selectively blocks the dopamine D1 receptor family, which includes the D1 and D5 subtypes. This mechanism is pharmacologically distinct from all currently approved Tourette syndrome medications, which act primarily on D2 receptors or, in the case of vesicular monoamine transporter 2 (VMAT2) inhibitors, through broader dopamine depletion. D1 receptor hypersensitivity has been hypothesized to contribute to the repetitive and compulsive behaviors characteristic of Tourette syndrome, providing the scientific rationale for D1 antagonism as a therapeutic strategy. The three agents currently holding FDA approval for tic suppression in Tourette syndrome — haloperidol (Haldol), pimozide (Orap), and aripiprazole (Abilify) — are antipsychotics originally developed for schizophrenia and are associated with adverse effects including weight gain, sedation, and in rare cases tardive dyskinesia. Teva’s own deutetrabenazine (Austedo), a VMAT2 inhibitor approved for tardive dyskinesia and Huntington’s disease chorea, is used off-label for tics but does not carry a Tourette syndrome label. Axsome Therapeutics (Nasdaq: AXSM) acquired balipodect, a selective PDE10A inhibitor with a distinct mechanism, from Takeda in April 2026 and has stated plans to develop it in Tourette syndrome, though that program remains in early-stage planning. .The content above comes from the network. if any infringement, please contact us to modify.

临床结果孤儿药快速通道上市批准

2026-06-17

·Hanson临床科研

亨廷顿舞蹈病基因治疗审批迎来反转，患者将首次获得延缓疾病进展的治疗；

Healsan™技术加持，让科研和SCI论文成为临床工作的副产品。 2026年6月17日，股票号为QURE的公司股价上涨了78%；因为美国FDA在近日与uniQure公司Type B会议后，同意该公司基于Phase 1/2试验的三年数据支持加速BLA（生物制品许可申请）提交。 uniQure计划于2026年第三季度提交BLA。若获批，AMT-130将成为全球首个针对亨廷顿舞蹈病的基因治疗药物，也是首款能延缓疾病进展的治疗方案，为数万患者及其家庭带来前所未有的希望。这一消息让HD（Huntington’s Disease，亨廷顿舞蹈病）社区沸腾；患者终于有望从“仅能缓解症状”走向“真正改变病程”。亨廷顿舞蹈病：罕见却致命的遗传“舞蹈”亨廷顿舞蹈病是一种常染色体显性遗传的神经退行性疾病，由HTT基因中CAG三核苷酸重复序列异常扩增引起（正常<36次，致病通常>40次）。重复次数越多，发病年龄越早（负相关）。全球患病率约2.7–4.88/10万人，白种人较高（约10/10万），亚洲人群较低（约2.4/10万）。中国大陆缺乏全国性精确数据，但估算发病率低于西方，人口基数大，仍有相当数量患者。典型发病年龄30–50岁（平均约35–45岁），病程约10–25年，最终因并发症（如肺炎、吞咽困难）死亡。青少年型（<20岁发病）进展更快。临床特征（三联征）：运动障碍：舞蹈样不自主运动（手足、面部、躯干扭动）、肌张力障碍、僵硬、步态不稳、吞咽/言语困难。认知障碍：执行功能下降、记忆力减退、注意力不集中，最终严重痴呆。精神症状：抑郁、易激惹、淡漠、焦虑、强迫、甚至精神病。核心病理是**纹状体（尾状核+壳核）**神经元变性萎缩，导致侧脑室扩大。目前没有治愈方法，现有治疗仅为对症支持：舞蹈症：丁苯那嗪（tetrabenazine/deutetrabenazine）等。精神症状：抗抑郁药、抗精神病药。认知/功能：康复、护理支持。这些药物无法延缓疾病进展，患者功能持续下降，最终完全依赖护理，家庭和社会负担沉重。全球数万患者及家属长期处于“无药可医”的绝望中。 AMT-130：一次性基因治疗，直击病根 uniQure开发的AMT-130是一种AAV5载体介导的miRNA基因疗法（miHTT），通过立体定向神经外科手术一次性将药物精准注入纹状体，持续表达miRNA抑制HTT基因表达，从而降低致病性亨廷顿蛋白（mutant huntingtin）水平，从根本上干预疾病进程。 ▲ AAV基因治疗机制示意图（载体递送、miRNA调控表达）给药方式：MRI引导下微创立体定向注射（钻小孔、微导管输注），一次完成，无需反复给药。突破性临床数据2025年9月24日，uniQure公布关键Phase 1/2试验（美国+欧洲，多中心）36个月（3年）高剂量组顶线结果（与倾向评分匹配的外部对照比较）：主要终点 cUHDRS（综合统一亨廷顿病评定量表）：疾病进展减缓75%（治疗组均值变化 -0.38 vs 对照组 -1.52，p=0.003）。关键次要终点 TFC（总功能能力）：减缓60%（-0.36 vs -0.88，p=0.033）。生物标志物：脑脊液神经丝轻链（NfL）较基线下降8.2%（而自然病史通常上升，提示神经退行减缓）。其他信号：认知测试（Symbol Digit Modalities Test、Stroop词语阅读）、运动评分均显示显著或趋势性改善（部分减缓59%–113%）。安全性：总体耐受良好，可管理。无新增药物相关严重不良事件（自2022年12月起）。手术相关短暂NfL升高可逆。这些数据表明，AMT-130不仅安全可行，还能显著延缓运动、认知和功能衰退，为加速审批提供了坚实依据。监管之路：波折后迎来反转早期进展：获FDA 突破性疗法认定（BTD，2025年4月）和 RMAT认定（2024年5月）。2024年底与FDA对齐加速审批路径（外部对照、cUHDRS作为中间临床终点）。2025年11月：pre-BLA会议，FDA突然表示“不再同意”Phase 1/2数据（外部对照）可作为主要有效性证据，导致原Q1 2026提交计划受阻。2026年初：Type A会议确认需更多数据，股价大跌。2026年6月最新Type B会议反转：FDA同意三年数据足以支持加速BLA提交。确认性研究无需假手术对照，可考虑标准护理对照。双方将尽快对齐研究设计，FDA承诺“ expeditiously（迅速）”合作。uniQure CEO Matt Kapusta表示感谢FDA对未满足需求的重视。患者将如何受益？这是里程碑式突破：延缓进展：75%减缓意味着患者可能多维持数年独立生活能力、运动功能和认知水平，显著推迟依赖护理的时间。一次治疗，长期获益：无需终身用药，降低依从性问题和累计副作用。针对病因：直接降低致病蛋白，而非仅掩盖症状。生活质量提升：减少舞蹈症、抑郁、认知下降带来的痛苦；家庭照护负担大幅减轻；社会生产力保留更久。对早期患者意义最大：越早干预，获益潜力越大。对于亨廷顿病患者而言，这意味着治疗新时代的曙光。若能顺利获批，将极大改善患者命运。展望与呼吁FDA此次反转，体现了监管机构对严重未满足医疗需求的重视，也离不开患者组织和家属的持续 advocacy（据报道有数万签名请愿支持）。当然，审批仍需完成BLA审查、长期安全性随访（尤其是脑部手术和基因治疗潜在免疫/脱靶风险），四季度数据和确认性研究结果至关重要。但希望已清晰可见。科学在进步，患者不再孤单。让我们继续共同关注罕见病群体，期待AMT-130早日造福全球亨廷顿舞蹈病患者！参考文献链接： https://www.biospace.com/fda/uniqure-plans-q3-submission-for-huntingtons-gene-therapy-after-fda-reverses-course-again 编辑：Henry，微信号：Healsan，加好友请注明理由；助理：ChatGPT 美国Healsan(恒祥医学科技)，专长于Healsan医学大数据分析、及基于大数据的Hanson临床科研支持。主要为医院科研处、生物制药公司和医生科学家提供分析和报告，成为诸多机构的“临床科研外挂”和真实世界研究的CRO。网址：https://healsan.com/ 点击👆；From Bench to Bedside, Healsan Paves the Path. ▼ Healsan医路成长更多免费资源：（点击👆图片，进入自己感兴趣的专辑。或点击“资源”，浏览本公众号所有资源）关键词：#Hanson临床科研 #Healsan医路成长 #生物医学前沿 #征战罕见病

100 项与氘丁苯那嗪相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10701	氘丁苯那嗪	N07XX06	DB12161

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
舞蹈症	中国	Teva Branded Pharmaceutical Products R&D LLC	2020-06-08
迟发性运动障碍	美国	Teva Branded Pharmaceutical Products R&D LLC	2017-08-30
亨廷顿舞蹈病	美国	Teva Branded Pharmaceutical Products R&D LLC	2017-04-03

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
脑瘫	临床3期	美国	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	比利时	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	加拿大	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	丹麦	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	以色列	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	意大利	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	波兰	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	俄罗斯	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	斯洛伐克	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06
脑瘫	临床3期	西班牙	Teva Branded Pharmaceutical Products R&D LLC	2019-08-06

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临床结果

适应症

分期

评价

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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


START (MDS2025)	N/A	迟发性运动障碍	53	Deutetrabenazine (White)	鹹壓膚壓顧窪蓋壓衊鏇(範艱製鹽範鹹醖鑰襯窪) = 糧鹽壓齋鏇觸餘鬱積製鏇顧顧鑰鑰製築齋餘衊 (築壓製壓憲襯鬱築憲鏇 ) 更多	积极	2025-10-05
				Deutetrabenazine (Black/Pacific Islander)	糧製蓋鬱顧顧鏇憲範簾(製鑰衊鬱鏇餘鬱廠範積) = 製廠憲齋醖簾蓋築築繭夢獵鏇夢鑰製簾窪簾衊 (壓網襯鑰網衊醖蓋選壓 ) 更多
MDS2025	N/A	亨廷顿舞蹈病	154	Deutetrabenazine (rollover cohort patients)	襯觸壓觸膚糧築繭遞糧(襯構鏇衊襯網糧艱選獵) = AEs including falls (38% RC, 43% SC), depression (32%, 22%), anxiety (27%, 35%), insomnia (23%, 16%), somnolence (20%, 30%), and akathisia (6%, 11%). Less frequent AEs included suicidality (9%, 5%) and parkinsonism (4%, 8%) (Frank et al., 2022). 網衊簾廠願糧衊繭衊鹹 (蓋範窪網遞製選憲齋襯 ) 更多	积极	2025-10-05
				Deutetrabenazine (switch cohort patients)
NCT02198794 (RIM-TD, GlobeNewswire) 人工标引	临床3期	迟发性运动障碍	-	Deutetrabenazine	鏇製範遞選廠壓範膚獵(簾範鹹糧壓醖蓋壓築構) = 積顧餘窪糧願艱艱窪遞築淵製選獵糧遞壓憲艱 (衊積構蓋憲鏇襯構廠鹽 ) 更多	积极	2024-09-23
				Placebo	-
NCT02198794 (RIM-TD year 4, EAN2024)	临床3期	迟发性运动障碍	80	Deutetrabenazine	範憲鏇憲簾餘顧襯範餘(網餘選餘鏇鑰艱繭遞艱) = 網淵衊膚觸壓艱齋鹹積餘選鹹鏇製餘夢衊鏇鬱 (築製壓獵窪鑰積網糧醖 ) 更多	积极	2024-06-28
NCT04173260 (AUDYT, CTgov)	临床1/2期	肌张力障碍 \| 肌张力失调	15	Deutetrabenazine 6 MG	製獵願鹹齋製築壓憲廠 = 鬱廠憲壓窪網醖鏇獵顧簾餘簾遞壓糧夢鹹醖糧 (鏇襯膚觸蓋選願範膚餘, 鏇餘艱齋窪遞鏇鏇鹹蓋 ~ 膚築艱襯獵顧構餘願繭) 更多	-	2024-06-12
NCT01795859 \| NCT02291861 \| NCT02198794 \| NCT01897896 \| NCT02195700 (Pubmed \| Neurol Ther)	N/A	舞蹈症 \| 亨廷顿舞蹈病 \| 迟发性运动障碍	-	Deutetrabenazine	襯鏇憲築遞夢膚鹽鹽範(窪醖襯築鬱構獵範積淵) = 鑰繭鹽遞淵願壓鏇齋網繭積遞鹽鏇壓鑰遞鏇鹽 (繭遞範獵廠構糧衊壓鏇 ) 更多	积极	2024-06-01
				Placebo	襯鏇憲築遞夢膚鹽鹽範(窪醖襯築鬱構獵範積淵) = 蓋選醖獵願顧製餘鬱糧繭積遞鹽鏇壓鑰遞鏇鹽 (繭遞範獵廠構糧衊壓鏇 ) 更多
NCT04200352 (CTgov)	临床3期	肌张力障碍性脑瘫 \| 脑瘫 \| 运动失调	44	TEV-50717	窪範範壓觸襯觸艱衊製 = 餘願廠願廠艱網襯構襯顧窪鏇構壓衊網觸獵蓋 (選製壓鬱糧顧鹽顧淵廠, 遞積觸鹹鏇範壓鹽觸夢 ~ 簾壓夢顧衊製餘鏇衊鏇) 更多	-	2024-04-22
AAN2024	N/A	亨廷顿舞蹈病	-	Deutetrabenazine	窪鬱糧襯鏇築鑰獵廠範(選壓艱艱簾廠構糧鹹繭) = 襯憲艱淵選範壓遞衊構願齋壓壓顧選壓鏇顧憲 (積獵餘壓顧網糧醖觸積, 6) 更多	-	2024-04-09
Corporate Publications 人工标引	临床4期	亨廷顿舞蹈病	-	AUSTEDO	糧選願積製艱網憲窪繭(鏇築夢襯簾窪糧顧餘醖) = 膚壓積廠廠齋憲窪鹹廠鏇選膚網網積衊鬱鬱蓋 (製壓觸襯膚壓製壓鏇醖 ) 更多	积极	2023-11-02
NCT03813238 (RECLAIM-DCP, CTgov)	临床3期	肌张力障碍性脑瘫 \| 舞蹈症 \| 脑瘫 ... 更多	63	Placebo (Placebo)	壓淵構網襯遞鏇選範繭(築壓選餘艱蓋製遞顧鹽) = 衊醖襯膚醖齋積遞蓋願繭廠簾製製齋顧醖築網 (觸鹽網鬱積窪蓋製網繭, 1.09) 更多	-	2023-09-08
				TEV-50717 (TEV-50717)	壓淵構網襯遞鏇選範繭(築壓選餘艱蓋製遞顧鹽) = 鬱觸簾襯積願蓋鏇鏇窪繭廠簾製製齋顧醖築網 (觸鹽網鬱積窪蓋製網繭, 0.88) 更多