This research study is studying troriluzole as a possible treatment for recurrent glioblastoma.
The name of the study drug involved in this research study is:
-Troriluzole (a tripeptide prodrug of riluzole)

开始日期2025-02-19

申办/合作机构

Dana-Farber Cancer Institute, Inc.

[+2]

CTR20213162

/ Completed临床1期

一项在中国健康受试者中评Troriluzole（BHV-4157）280mg单次和多次给药后的安全性、耐受性和药代动力学特征的I期、随机、双盲、安慰剂对照研究

主要目的：评估在中国健康受试者中单次（280 mg）和多次（280 mg/日，连续5天）给药Troriluzole后的安全性和耐受性。次要目的：评估在中国健康受试者中单次（280 mg）和多次（280 mg/日，连续5天）给药Troriluzole后利鲁唑的药代动力学（PK）特征。

开始日期2022-03-03

申办/合作机构

标新有限公司

[+2]

NCT04899921

/ Terminated临床2期IIT

A Blinded, Randomized Phase 2 Study of Troriluzole in Combination With Ipilimumab and Nivolumab in Patients With Melanoma Brain Metastases Previously Treated With Anti-PD-1 Therapy

The purpose of this research is to test the safety and effectiveness of the investigational combination of Troriluzole, ipilimumab, and nivolumab, and to learn whether this combination works in treating melanoma that has spread to the brain.

开始日期2021-06-30

申办/合作机构

Dana-Farber Cancer Institute, Inc.

[+1]

100 项与 Trigriluzole 相关的临床结果

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100 项与 Trigriluzole 相关的转化医学

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100 项与 Trigriluzole 相关的专利（医药）

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项与 Trigriluzole 相关的文献（医药）

2025-04-01·Neurology and Therapy

Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia

Article

作者: Beiner, Melissa Wolfe ; L'Italien, Gilbert ; Coric, Vlad ; Subramony, Sub ; Synofzik, Matthis ; Popoff, Evan ; Powell, Lauren C ; Potashman, Michele H ; Schmahmann, Jeremy ; Mackenzie, Ainsley

INTRODUCTION:

The Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with Friedreich ataxia. We provide evidence of the psychometric validity of the FARS-ADL in two cohorts of patients with spinocerebellar ataxia (SCA).

METHODS:

Using data from a cohort of real-world subjects with SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), comprising a subset of patients with the SCA3 genotype (n = 89), the psychometric measurement properties and minimal change thresholds of the FARS-ADL were examined.

RESULTS:

Ceiling effects for the FARS-ADL were absent within the MGH cohort while floor effects were observed for eight of nine items. Excellent internal consistency reliability was observed (α_total = 0.88; α_{items-removed} = 0.86-0.87), and item-to-total correlations were acceptable (r = 0.55-0.89 per item). Convergent and divergent validity were supported with strong correlations demonstrated between FARS-ADL and scales measuring similar concepts (Neuro-QOL [Upper], Neuro-QOL [Lower], PROM-ADL, PROM-PHYS, and FARS-FUNC; all P < 0.001) and weaker correlations shown between measures of differing constructs. A two- to three-point threshold for meaningful changes was supported as 0.5 × SD = 2.43, SEM = 2.19. Mean changes from baseline for subjects classified as "improved," "no change," or "deteriorated" were -0.54, 0.22, and 1.47, respectively. Similar trends were observed in the Study 206 all-SCA and SCA3 cohorts.

CONCLUSION:

Psychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3.

TRIAL REGISTRATION:

ClinicalTrials.gov identifier, NCT03701399 (Study 206).

2025-01-01·JOURNAL OF NEUROCHEMISTRY

Troriluzole rescues glutamatergic deficits, amyloid and tau pathology, and synaptic and memory impairments in 3xTg‐AD mice

Article

作者: Perman, Savannah ; Coric, Vladimir ; Pfitzer, Jeremiah ; Reed, Miranda N. ; Suppiramaniam, Vishnu ; Pinky, Priyanka D. ; Redmon, Emma ; Gramlich, Michael W. ; Qureshi, Irfan A. ; Cmelak, Luca

Abstract:

Alzheimer's disease (AD) is a neurodegenerative condition in which clinical symptoms are highly correlated with the loss of glutamatergic synapses. While later stages of AD are associated with markedly decreased glutamate levels due to neuronal loss, in the early stages, pathological accumulation of glutamate and hyperactivity contribute to AD pathology and cognitive dysfunction. There is increasing awareness that presynaptic dysfunction, particularly synaptic vesicle (SV) alterations, play a key role in mediating this early‐stage hyperactivity. In the current study, we sought to determine whether the 3xTg mouse model of AD that exhibits both beta‐amyloid (Aβ) and tau‐related pathology would exhibit similar presynaptic changes as previously observed in amyloid or tau models separately. Hippocampal cultures from 3xTg mice were used to determine whether presynaptic vesicular glutamate transporters (VGlut) and glutamate are increased at the synaptic level while controlling for postsynaptic activity. We observed that 3xTg hippocampal cultures exhibited increased VGlut1 associated with an increase in glutamate release, similar to prior observations in cultures from tau mouse models. However, the SV pool size was also increased in 3xTg cultures, an effect not previously observed in tau mouse models but observed in Aβ models, suggesting the changes in pool size may be due to Aβ and not tau. Second, we sought to determine whether treatment with troriluzole, a novel 3rd generation tripeptide prodrug of the glutamate modulator riluzole, could reduce VGlut1 and glutamate release to restore cognitive deficits in 8‐month‐old 3xTg mice. Treatment with troriluzole reduced VGlut1 expression, decreased basal and evoked glutamate release, and restored cognitive deficits in 3xTg mice. Together, these findings suggest presynaptic alterations are early events in AD that represent potential targets for therapeutic intervention, and these results support the promise of glutamate‐modulating drugs such as troriluzole in Alzheimer's disease. image

2024-08-01·Neurology and Therapy

Video-Based Kinematic Analysis of Movement Quality in a Phase 3 Clinical Trial of Troriluzole in Adults with Spinocerebellar Ataxia: A Post Hoc Analysis

Article

作者: Perlman, Susan ; Maclaine, Grant D H ; L'Italien, Gilbert J ; Coric, Vladimir ; Khera, Rohan ; Potashman, Michele H ; Schmahmann, Jeremy D ; Beiner, Melissa W ; Oikonomou, Evangelos K

INTRODUCTION:

Traditional methods for assessing movement quality rely on subjective standardized scales and clinical expertise. This limitation creates challenges for assessing patients with spinocerebellar ataxia (SCA), in whom changes in mobility can be subtle and varied. We hypothesized that a machine learning analytic system might complement traditional clinician-rated measures of gait. Our objective was to use a video-based assessment of gait dispersion to compare the effects of troriluzole with placebo on gait quality in adults with SCA.

METHODS:

Participants with SCA underwent gait assessment in a phase 3, double-blind, placebo-controlled trial of troriluzole (NCT03701399). Videos were processed through a deep learning pose extraction algorithm, followed by the estimation of a novel gait stability measure, the Pose Dispersion Index, quantifying the frame-by-frame symmetry, balance, and stability during natural and tandem walk tasks. The effects of troriluzole treatment were assessed in mixed linear models, participant-level grouping, and treatment group-by-visit week interaction adjusted for age, sex, baseline modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA), and time since diagnosis.

RESULTS:

From 218 randomized participants, 67 and 56 participants had interpretable videos of a tandem and natural walk attempt, respectively. At Week 48, individuals assigned to troriluzole exhibited significant (p = 0.010) improvement in tandem walk Pose Dispersion Index versus placebo {adjusted interaction coefficient: 0.584 [95% confidence interval (CI) 0.137 to 1.031]}. A similar, nonsignificant trend was observed in the natural walk assessment [coefficient: 1.198 (95% CI - 1.067 to 3.462)]. Further, lower baseline Pose Dispersion Index during the natural walk was significantly (p = 0.041) associated with a higher risk of subsequent falls [adjusted Poisson coefficient: - 0.356 [95% CI - 0.697 to - 0.014)].

CONCLUSION:

Using this novel approach, troriluzole-treated subjects demonstrated improvement in gait as compared to placebo for the tandem walk. Machine learning applied to video-captured gait parameters can complement clinician-reported motor assessment in adults with SCA. The Pose Dispersion Index may enhance assessment in future research. TRIAL REGISTRATION-CLINICALTRIALS.

GOV IDENTIFIER:

NCT03701399.

104

项与 Trigriluzole 相关的新闻（医药）

2025-10-19

·猎药零零囧

2025小脑萎缩新药前沿：神经退行性疾病领域迎来历史性突破，脊髓小脑性共济失调（SCA）治疗进入商业化元年

首款药物呼之欲出，多靶点管线竞速进入2025年，神经退行性疾病领域迎来了诸多激动人心的变革，而长期以来被视为"不可治疗"的小脑萎缩及其相关疾病，特别是脊髓小脑性共济失调（Spinocerebellar Ataxia, SCA），正处于突破的前夜。对于全球数以万计的患者、研发人员及投资者而言，今年注定是具有里程碑意义的一年。多款创新疗法在临床开发中取得了关键进展，其中一款药物更有望在年内成为首个获批上市的SCA治疗药物，打破该领域无药可医的困境。核心发现摘要首款SCA治疗药物Troriluzole已获FDA优先审评资格，预计2025年第三季度做出最终决定反义寡核苷酸（ASO）疗法成为精准靶向SCA亚型的主流策略基因编辑和干细胞疗法等前沿技术为长期治疗提供新希望SCA治疗领域从高风险无回报转向具有明确市场潜力的赛道首款SCA治疗药物Troriluzole静待FDA裁决 1.1 药物机制与临床地位 Troriluzole是一种第三代前体药物，在体内可转化为利鲁唑（Riluzole），通过调节神经系统中的谷氨酸水平来发挥作用。谷氨酸是中枢神经系统中主要的兴奋性神经递质，其过度活化被认为是导致SCA等神经退行性疾病中神经元损伤和死亡的关键机制之一。作用机制详解： Troriluzole的设计旨在提供更优的药代动力学特性和更好的耐受性，相比传统利鲁唑，它具有以下优势：提高生物利用度，减少给药频率改善血脑屏障穿透能力降低潜在副作用发生率延长半衰期，优化给药方案 1.2 关键里程碑事件优先审评资格美国食品药品监督管理局（FDA）已受理Troriluzole用于治疗SCA的新药申请（NDA），并授予了优先审评资格（Priority Review）。这本身就表明了FDA对该药物潜在临床价值的高度认可，也意味着审评周期将从标准的10个月缩短至6个月。 PDUFA日期根据公开信息，FDA预计将在2025年第三季度对Troriluzole的上市申请做出最终决定。这一时间表为市场和投资者提供了明确的预期时间线。 III期临床试验关键III期临床试验显示积极顶线数据，证明药物在延缓疾病进展方面具有显著疗效，为后续监管审批提供了强有力的支持。 1.3 专家深度解读 "Troriluzole的获批将是历史性的。它不仅将成为全球首款被正式批准用于治疗SCA的药物，更重要的是，它将彻底改变整个小脑萎缩领域的治疗格局和研发信心。" 多维度影响分析：对于患者意味着终于有了经循证医学验证的、能够延缓疾病进展的治疗选择。这将显著改善患者的生活质量，延长独立生活能力。对于研发成功上市的案例将极大地激励后续企业和研究机构投入资源，加速其他创新靶点和疗法的开发。其关键III期临床试验的积极顶线数据也为后续临床试验的设计提供了宝贵的参考。对于投资标志着SCA从一个高风险、无商业回报的领域，转变为一个具有明确市场潜力和增长前景的赛道，预计将吸引更多资本注入。据估计，SCA药物市场规模将在2025年后达到数十亿美元级别。临床管线中的核心力量与多元化布局在Troriluzole冲刺上市的同时，2025年的临床管线也展示了小脑萎缩疗法开发的多样性和深度，特别是以基因为靶点的精准疗法正成为研发主流。 2.1 反义寡核苷酸（ASO）疗法：精准靶向致病基因 ASO疗法通过设计与特定致病基因mRNA互补的短核酸链，实现对异常蛋白表达的精准"沉默"，是治疗遗传性SCA的理想策略。 VO659 (Vico Therapeutics) 针对SCA1型和SCA3型 I/IIa期开放标签试验在2025年1月发布更新，显示出良好的安全性和靶向抑制潜力，目前研究仍在进行中。 SRP-1004 (Sarepta Therapeutics) 专为SCA2型开发计划在2025年第三季度启动其I期临床研究的第二个队列入组，表明该项目正在按计划稳步推进。 Cure Rare Disease ASO 治疗包括SCA3在内的多种SCA 2025年5月，FDA授予该药物孤儿药资格（ODD），为其后续开发提供政策支持和市场独占期。 ASO技术优势：高特异性：能够精确靶向特定基因突变可逆性：通过调整剂量可调节药物效果递送系统成熟：鞘内注射技术已广泛应用临床数据支持：多个ASO药物已获批上市 2.2 临床开发中的挑战与教训渤健（Biogen）BIBI132项目终止针对SCA3的候选药物BIBI132的I期临床试验，原计划于2025年完成，但最终被终止。这一挫折凸显了CNS药物开发的高风险性，即便是行业巨头也面临巨大挑战。失败案例提供的宝贵经验：靶点选择：需要更深入理解疾病特异性机制递送方式：优化药物穿透血脑屏障的能力安全性评估：加强早期安全性监测和风险管理剂量确定：建立更精准的起始剂量和滴定方案 2.3 其他创新机制的探索 SLS-005 (海藻糖注射液, Seelos Therapeutics) 作用机制是通过稳定蛋白质结构和增强细胞自噬来清除异常蛋白聚集。评估其治疗SCA的II期STRIDES研究已于2024年下半年完成，其详细数据分析和结果公布是2025年学界和业界关注的焦点之一。作用机制详解：稳定蛋白质折叠：防止错误折叠蛋白积累促进细胞自噬：清除受损细胞器和错误折叠蛋白减轻内质网应激：降低细胞毒性反应改善线粒体功能：增强能量代谢未来展望：前沿靶点与下一代治疗技术除了已进入临床阶段的药物，基础研究的持续深入也为未来的小脑萎缩治疗开辟了更多可能性。 3.1 基因编辑：从"沉默"到"修复"的终极疗法以CRISPR-Cas9为代表的基因编辑技术，被视为根治遗传性神经疾病的终极解决方案。研究显示，在弗里德莱希共济失调（FRDA，一种与小脑萎缩相关性极高的遗传病）的小鼠模型中，通过基因编辑技术切除异常重复序列已取得成功。技术挑战与解决方案：递送效率：开发新型AAV载体提高靶向性脱靶效应：优化sgRNA设计和Cas9变体免疫原性：降低免疫排斥反应长期表达：确保基因编辑效果持久性尽管将其应用于人体的临床转化仍面临递送效率和脱靶效应等挑战，但这无疑代表了未来10-15年的重要发展方向。 3.2 干细胞疗法：修复受损神经元的潜力干细胞疗法旨在通过移植新的神经干细胞或祖细胞，替代或修复小脑中受损的神经元。2025年的研究进展表明，干细胞在动物模型中展现出改善运动功能和延缓神经退变的潜力。临床转化关键挑战：细胞存活率：提高移植细胞的长期存活率分化控制：精确控制细胞分化为所需神经元类型功能整合：确保新神经元与现有神经网络有效连接长期安全性：排除肿瘤形成和免疫排斥风险 3.3 新型分子靶点与机制研究虽然2025年尚未在《Nature》、《Science》等顶级期刊上出现直接针对小脑萎缩新靶点的突破性论文报道，但对相关致病蛋白如ATXN家族的研究正不断深入。 ATXN1-CIC蛋白复合物功能障碍研究揭示了ATXN1与CIC蛋白相互作用异常导致神经元功能障碍的机制，为开发靶向该复合物的小分子抑制剂提供了理论基础。 ATXN2蛋白相分离特性发现ATXN2蛋白在特定条件下会发生异常相分离，形成有毒聚集体，这一发现为开发阻止蛋白聚集的药物提供了新思路。未来研究方向：开发靶向ATXN1-CIC相互作用的小分子设计阻止ATXN2相分离的化合物探索RNA结合蛋白的调控机制研究线粒体功能障碍的干预策略结论与专家洞察综上所述，2025年是小脑萎缩创新药物研发从量变到质变的关键转折点。我们对当前形势和未来趋势进行综合分析：首药上市在即，市场格局将被重塑 Troriluzole若能如期获批，将不仅为SCA患者带来希望，更会激活整个领域的研发和投资热情，标志着小脑萎缩治疗进入商业化元年。精准医疗成为主流，ASO管线梯队形成针对不同SCA亚型的ASO疗法构成了目前最有潜力的在研管线。Vico、Sarepta等公司的稳步推进，预示着未来几年将有更多基因靶向药物进入中后期临床。技术平台多元化，下一代疗法蓄势待发虽然基因编辑和干细胞疗法等前沿技术短期内尚难实现临床转化，但其展现出的巨大潜力正吸引着持续的研究投入，它们是解决小脑萎缩这一顽疾的长期希望所在。战略建议研发人员与立项者密切关注Troriluzole的审评结果及其上市后的真实世界数据重点布局具有明确遗传靶点的SCA亚型优先考虑ASO和RNAi等技术平台的项目关注多靶点联合治疗策略的开发前瞻性投资者开始关注在基因编辑递送技术和干细胞临床转化方面取得突破性进展的早期公司投资具有差异化技术平台的企业布局SCA诊断工具和伴随诊断开发关注数字医疗和远程监测技术在疾病管理中的应用 "小脑萎缩的'冰山'正在融化，一个充满机遇与挑战的新时代已经到来。随着首款治疗药物的临近获批，以及多条创新管线的稳步推进，我们有理由相信，不久的将来，小脑萎缩患者将迎来真正的治疗曙光。" 风险声明本文基于公开信息整理，不构成投资建议。报告内容仅用于学术研究和行业参考，不涉及任何投资决策建议。文中所有数据和信息均来源于可公开获取的资料，包括但不限于：已发表的学术论文和临床试验报告监管机构（如FDA）的公开文件上市公司财报和投资者关系披露行业媒体和专业新闻报道如有错误或不实报道，请留言联系作者纠正。感谢您的阅读与支持。参考文献 1. CNS创新药系列报告（二）：检测革新和新药突破双因素共振，阿尔茨海默病诊疗双螺旋上升（治疗篇）. 太平洋证券股份有限公司证券研究报告.[2024-01-20] 2. 2025年内科学研究新进展[2025-03-14] 3. Cerebellar ataxia research update. Dr Julie et al. 4. Demonstration of Cerebellar Atrophy in Neuroacanthocytosis of 2 Siblings. T. Katsume et al. 5. Advances in physiological and clinical relevance of hiPSC-derived brain models for precision medicine pipelines. Negin Imani Farahani et al.[2025-01-06] 6. SUFU 2025 Winter Meeting. Westin Rancho Mirage Golf Resort & Spa, Rancho Mirage, CA.[2025-02-26] 7. 阿尔茨海默病药物研发管线：2025. 美国多个顶尖研究机构.[2025-06-09] 8. Selected Innovative Medicines in Development as of January 22, 2025 9. Mind over matter: The growing momentum of CNS innovation. MARKUS GORES et al. 10. Pesquisa e saúde III: a importância da iniciação científica. Mônica Jachetti Maciel et al.[2025] 11. 2023年15款里程碑新药批准[2024-02-08] 12. 研究指抑制劑阻腦細胞受攻擊助研小腦萎縮症新藥. TVB News.[2019-05-06] 13. 首都医学科学创新中心[2025-07-04] 14. Drug Development & Delivery[2023-05-23] 15. UCB presents key data from Alzheimer’s and Parkinson’s disease research programs at AD/PD 2025. UCB, Brussels, Belgium.[2025-04-01] 16. 2025年药物研发领域新进展. 幻目.[2025-03-03] 17. CNSA Contemporary Neurology 2025 18. 2025 NIH Alzheimer’s Disease and Related Dementias Research Progress Report: Advances and Achievements. National Institutes of Health. 19. Top 10 Emerging Technologies of 2025. World Economic Forum, Centre for the Fourth Industrial Revolution.[2025-06] …………………… 陪伴是最长情的告白为你推送最实用的资讯识别二维码关注我们喜欢就点个赞吧

优先审批申请上市临床3期细胞疗法

2025-10-14

·药时空

年终冲刺！18款新药待审批，2025年FDA最终成绩单将如何？

2025年，美国FDA的新药审批节奏明显放缓，全年批准数量或将创下近三年以来的新低。尽管批准数量有所放缓，但今年已获批的新药在肿瘤精准治疗、罕见病治疗以及药物作用机制多元化等方面，展现出显著的创新质量。本文将从批准趋势、重点领域突破、待批重磅药物等角度，系统梳理2025年FDA新药批准的总体图景。 01 审批数量“断崖式”下降，创近三年新低根据Endpoints News的统计，FDA今年迄今已批准42种新药和生物制剂，剩余约16项审批决定将在2026年前完成。不过，即便剩余申请全部获批，2025年全年总量也仅能达到58种左右，远低于2023年的80种和2024年的69种。将时间线继续向前推，2020年-2022年，FDA分别批准了61种、63种以及51种新药和生物制剂。由此看来，2025年或将成为近五年以来，FDA审批数量次低水平的一年。尽管审批数量下降，但数量并不完全等同于创新质量。真正的价值在于其是否解决了未被满足的临床需求。例如，2025年前三季度批准的32款新药中，包含多款First-in-class（首创机制）和孤儿药，显示出“少而精”的特点。值得注意的是，FDA发言人明确表示，药物审评与审批未受民主党主导的政府停摆影响，相关工作仍在持续推进。不过，目前还不清楚数千名FDA工作人员的流失，是否在今年减慢审批进程方面发挥了作用。如果申请继续以目前的速度被延迟，今年的批准数量不太可能达到历史高位。早在7月，FDA专员Marty Makary在接受CNBC采访时表示，该机构“可能在2025年创下批准数量的新纪录”。FDA发言人并未对这一预测是否仍然有效发表评论。 02 肿瘤与罕见病：创新药物的主战场据FDA新药图鉴统计显示，2025年1-9月FDA共批准上市32款新药。按季度看，Q1季度共批准上市7款新药，Q2季度共获批9款新药，Q3季度共获批16款新药。按分子类型看，前三季度共获批小分子药物22款，生物大分子药物10款。其中，10款生物大分子药物中，包括5款单克隆抗体药物、2款抗体药物偶联物（ADC）、2款小核酸药物，以及1款双特异性抗体药物。按获批适应症看，包括肿瘤12款、罕见病11款、感染性疾病2款、眼部疾病2款、皮肤病2款、肺部、免疫和神经性疾病各1款。涉及新药认定包括孤儿药（18/32）、First-in-class（17/32）、突破性疗法（10/32）、首轮批准、优先审批及快速通道。此外，4款药物带黑框警告上市。总体而言，2025年FDA现批准的新药呈现出肿瘤治疗继续向精准化、免疫化与联合化深入；罕见病药物开发蓬勃，许多既往无药可治的疾病迎来了首款疗法；药物作用机制日益多元，技术平台丰富化等研发趋势。这些进展共同体现了医药研发从传统机制向前沿技术平台的拓展，为治疗提供了更多可能性。 03 年底冲刺：18款重磅新药待批尽管2025年全年审批量遇冷，但在今年剩余的两个多月，仍有18款新药等待FDA的最终审批。这些待批药物涵盖了眼科、心血管、代谢疾病、抗感染等多个领域，其中不乏备受关注的重磅产品。而这些药物的审评结果，将直接影响2025年最终的新药批准总量和创新成色。以下为今年年底待批的18款新药产品： 10月20日：Glaukos的Epioxa，这是一种用于圆锥角膜患者的角膜疗法，无需去除角膜上皮； 10月23日：Sydnexis的SYD-101，一种用于儿童近视进展的低剂量阿托品眼药水； 10月26日：拜耳的elinzanetant，一种用于缓解中度至重度潮热（由更年期引起）的神经激肽1和神经激肽3受体拮抗剂； 11月18日：Arrowhead Pharmaceuticals的plozasiran，一种用于治疗高甘油三酯水平的RNAi药物； 11月28日：大冢制药的sibeprenlimab，用于IgA肾病； 11月30日：Kura Oncology的ziftomenib，用于治疗携带核磷蛋白1突变的成人复发或难治性急性髓系白血病； 11月30日：Ascendis Pharma的TransCon CNP，一种C型利钠肽的前药，用于治疗软骨发育不全患者； 12月5日：Axogen的Avance神经移植，一种用于桥接断裂周围神经的现成人类神经异体移植； 12月12日：BioCryst的Orladeyo口服颗粒，用于治疗遗传性血管性水肿儿童； 12月13日：Milestone Pharmaceuticals的etripamil鼻喷雾剂，用于治疗阵发性室上性心动过速患者； 12月15日：Innoviva的zoliflodacin，一种用于治疗12岁及以上成人和儿童单纯性淋病的口服抗生素； 12月16日：GSK的depemokimab，用于两种哮喘适应症； 12月16日：Aldeyra的reproxalap，用于缓解干眼症的体征和症状； 12月26日：Cytokinetics的aficamten，用于治疗梗阻性肥厚型心肌病； 12月26日：Omeros的narsoplimab，用于治疗造血干细胞移植相关血栓性微血管病； 12月30日：Vanda Pharmaceuticals的tradipitant，用于治疗胃轻瘫；第四季度：Biohaven的troriluzole，用于治疗脊髓小脑性共济失调；第四季度：三菱田边制药的帕金森病治疗药物。总的来说，2025年FDA新药审批的“降温”，更多是周期性波动而非创新衰退。在研发成本高企、监管趋严、靶点竞争激烈的背景下，药企正从“追求数量”转向“追求质量”。尽管全年批准总数可能下降，但First-in-class药物、罕见病突破和精准治疗的进展，依然为患者带来了新的希望。参考资料： [1] FDA on pace for lowest number of new drug approvals since 2022.Endpoints News.October 13, 2025 03:30 PM EDTUpdated 04:01 PM FDA+. [2] 2025年：FDA已批准33款新药. 药事纵横. 2025-10-10. [3] 新药图鉴 | 2025年1-9月FDA批准上市32款新药可视化盘点，一图胜千言！FDA新药图鉴. 2025-10-01.

孤儿药优先审批抗体药物偶联物快速通道突破性疗法

2025-10-13

·EndPoints

FDA on pace for lowest number of new drug approvals since 2022

The FDA in 2025 is set to approve its lowest number of new drug and biologic applications since 2022, even as more than a dozen companies await the regulator’s decisions on their new molecular entities before the end of the year. The number of drug approvals isn’t always indicative of the quality of innovation or whether major strides are being made in hard-to-treat diseases. But it does provide an indication of R&D performance year-over-year. It’s also unclear whether losing thousands of FDA staff played a role in slowing down the approval process this year. The FDA has approved 42 new drugs and biologics so far this year, with about 16 decisions to go before 2026, according to an Endpoints News tally . In comparison, the regulator approved 69 new drugs and biologics in 2024, and 80 in 2023. But it was 2022 that had the fewest number of new approvals since 2020, with 51 new drugs and biologics approved. In 2020, the FDA approved 61 new drugs and biologics, and 63 in 2021. An FDA spokesperson told Endpoints News that “drug reviews and approvals are not affected by the Democrat-led shutdown and this work continues.” This year’s approval numbers likely won’t hit historic highs if applications continue to be delayed at the same rate as they have been recently . Back in July, FDA Commissioner Marty Makary told CNBC that the agency “could hit a record number of approvals” in 2025. The FDA spokesperson did not comment on whether that prediction still stands. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith! You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. Hello, Craig Smith. You’re an essential part of the biopharma world. Share your voice on this subscriber Pulse Survey to help shape our understanding of the industry. Results will be anonymous. At least 18 companies are still waiting for the FDA to decide whether to approve their new drug or biologic applications this year:

上市批准加速审批

100 项与 Trigriluzole 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Trigriluzole	-	DB15079

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
马查多-约瑟夫病	申请上市	美国	Biohaven Ltd.	2023-05-31
脊髓小脑性共济失调	申请上市	欧盟	Biohaven Ltd.	-
强迫症	临床3期	美国	Biohaven Pharmaceuticals, Inc.	2020-12-22
脊髓小脑性共济失调10型	临床3期	美国	Biohaven Pharmaceuticals, Inc.	2019-03-08
脊髓小脑性共济失调10型	临床3期	中国	Biohaven Pharmaceuticals, Inc.	2019-03-08
脊髓小脑性共济失调8型	临床3期	美国	Biohaven Pharmaceuticals, Inc.	2019-03-08
脊髓小脑性共济失调8型	临床3期	中国	Biohaven Pharmaceuticals, Inc.	2019-03-08
广泛性焦虑障碍	临床3期	美国	Biohaven Pharmaceuticals, Inc.	2019-02-19
阿尔茨海默症	临床3期	美国	Biohaven Pharmaceuticals, Inc.	2018-07-31
转移性黑色素瘤	临床2期	美国	Biohaven Pharmaceuticals, Inc.	2021-06-30

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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


NCT06529146 (BHV4157-206-RWE, Company_Website) 人工标引	N/A	脊髓小脑性共济失调	-	Troriluzole	膚醖積積顧餘鑰鹹憲鬱(壓鹽襯膚淵鹹蓋網夢範) = troriluzole 200 mg dosed orally in patients with SCA met the study's primary endpoint of change from baseline. 壓簾壓醖選範簾襯壓遞 (構觸構願願夢鹽襯願憲 ) 达到更多	积极	2025-02-11
				external control arm
NCT03701399 (Phase III Clinical Trial of Troriluzole, CTgov)	临床3期	脊髓小脑性共济失调10型 \| 马查多-约瑟夫病 \| 脊髓小脑性共济失调8型	299	troriluzole (Troriluzole)	衊糧夢製製淵壓蓋選襯(獵積鹹衊窪鏇鹽膚淵廠) = 膚遞積簾齋遞夢願鏇膚選衊壓積鏇製遞願衊壓 (膚鏇襯繭糧選顧範顧製, 0.19) 更多	-	2025-02-10
				Placebo+troriluzole (Placebo)	衊糧夢製製淵壓蓋選襯(獵積鹹衊窪鏇鹽膚淵廠) = 淵廠鹹願衊廠鑰艱範艱選衊壓積鏇製遞願衊壓 (膚鏇襯繭糧選顧範顧製, 0.18) 更多
NEWS 人工标引	临床3期	脊髓小脑性共济失调	-	Troriluzole	壓構夢壓積衊膚鹹觸壓(構鏇獵積鬱願觸夢網顧) = Biohaven said that the once-daily oral drug met the primary endpoint across all genotypes of SCA included in the study and that it showed statistically significant superiority after both one and two years compared to the external control groups. 遞鏇獵鹽顧選淵簾醖範 (衊夢選窪衊製鬱窪顧積 ) 达到更多	积极	2024-09-23
AAN2024	N/A	-	-	Troriluzole 200 mg	蓋選顧製願壓選繭膚鏇(簾願製鹽醖齋製築遞築) = values were 40% and 50% higher after troriluzole 100 mg and 280 mg, respectively vs oral riluzole 50 mg 鏇鏇鹽餘壓壓鹹醖鬱鏇 (簾糧鏇範衊網憲積淵觸 ) 更多	-	2024-04-09
				Troriluzole 280 mg
NCT03701399 (Phase III Clinical Trial of Troriluzole, AAN2024)	临床3期	脊髓小脑性共济失调	218	Troriluzole	壓齋襯遞壓壓淵製艱齋(衊壓壓選遞繭蓋觸齋顧): adj. interaction coefficient = 0.58 (95% CI, 0.14 ~ 1.03)	积极	2024-04-09
				Placebo
AAN2024	N/A	-	-	Troriluzole 140mg QD	窪網鬱鹽願鏇餘繭範膚(鹽範網醖網築選顧蓋壓) = 網網獵遞鬱鏇襯繭範網顧遞遞淵壓鏇積蓋製齋 (觸願製窪餘鏇構壓鹹簾 ) 更多	-	2024-04-09
				Troriluzole 200mg QD	窪網鬱鹽願鏇餘繭範膚(鹽範網醖網築選顧蓋壓) = 衊遞簾糧壓鹽糧艱築糧顧遞遞淵壓鏇積蓋製齋 (觸願製窪餘鏇構壓鹹簾 ) 更多
NCT04899921 (CTgov)	临床2期	转移性黑色素瘤	1	Ipilimumab+Troriluzole+Nivolumab (Ipilimumab + Nivolumab + Troriluzole [Phase I Dose Level 1])	廠餘醖鬱餘鬱遞蓋齋鹽(壓憲壓衊膚選窪餘選鏇) = 蓋鏇遞醖蓋衊醖餘糧選築積醖遞蓋觸襯網膚糧 (製築築壓鑰膚齋廠夢夢, 淵簾顧膚醖鑰鹽艱齋選 ~ 夢衊築齋糧襯願遞簾簾) 更多	-	2023-12-13
				Ipilimumab+Troriluzole+Nivolumab (Ipilimumab + Nivolumab + Troriluzole [Phase I Dose Level 2])	廠餘醖鬱餘鬱遞蓋齋鹽(壓憲壓衊膚選窪餘選鏇) = 鹹鏇顧顧夢選衊製廠鏇築積醖遞蓋觸襯網膚糧 (製築築壓鑰膚齋廠夢夢, 齋遞積積構鏇醖齋壓鏇 ~ 鏇製艱製簾獵獵餘遞網) 更多
NCT03605667 (T2 Protect AD, CTgov)	临床2期	阿尔茨海默症	350	Placebo oral capsule	鬱艱廠獵鹹糧範鬱衊顧(鬱積壓願鹽範獵蓋鏇襯) = 淵憲願簾蓋鑰簾淵鬱繭壓鬱製鹽製願範憲夢糧 (願夢網鹹窪願鬱網鏇齋, 觸顧鑰獵鏇網艱膚築簾 ~ 衊餘觸鑰夢網獵餘夢構) 更多	-	2023-12-06
NCT03299166 (CTgov)	临床2/3期	强迫症	426	Troriluzole (Troriluzole - Randomization Phase)	憲獵繭鬱製獵繭夢壓淵(鹽淵衊糧獵糧襯壓願壓) = 鏇遞夢網廠簾糧淵齋衊鹹齋製鏇簾鏇網餘壓製 (艱淵獵廠願製膚膚淵糧, 願艱蓋膚蓋淵醖繭鬱構 ~ 顧鬱願餘壓製繭夢夢鬱) 更多	-	2023-06-02
				placebo+troriluzole (Placebo - Randomization Phase)	憲獵繭鬱製獵繭夢壓淵(鹽淵衊糧獵糧襯壓願壓) = 蓋鹽廠淵繭淵鏇選積鏇鹹齋製鏇簾鏇網餘壓製 (艱淵獵廠願製膚膚淵糧, 製蓋夢顧範遞築壓廠繭 ~ 範構憲選餘積醖鑰夢觸) 更多
NCT03829241 (CTgov)	临床3期	广泛性焦虑障碍	881	Troriluzole (Troriluzole)	醖艱淵艱製淵廠鹽製顧(廠鏇觸遞鹹顧網廠廠築) = 淵醖壓築觸簾觸艱鑰壓簾衊壓鹽鹽餘憲繭鏇積 (製壓鏇願願齋淵簾鬱廠, 憲醖壓鬱衊顧鹹觸襯範 ~ 鹹餘膚糧艱範鬱鹹齋觸) 更多	-	2022-12-28
				Placebo+Troriluzole (Placebo)	醖艱淵艱製淵廠鹽製顧(廠鏇觸遞鹹顧網廠廠築) = 遞遞窪願餘齋膚築觸鑰簾衊壓鹽鹽餘憲繭鏇積 (製壓鏇願願齋淵簾鬱廠, 獵繭蓋願艱築範鹹鹽鑰 ~ 鬱憲齋鏇繭糧積壓淵艱) 更多