预约演示

更新于：2025-05-07

Cerebellar Ataxia

小脑共济失调

更新于：2025-05-07

基本信息

别名

ATAXIA CEREBELLAR、Ataxia、Ataxia cerebellar

+ [52]

简介

Incoordination of voluntary movements that occur as a manifestation of CEREBELLAR DISEASES. Characteristic features include a tendency for limb movements to overshoot or undershoot a target (dysmetria), a tremor that occurs during attempted movements (intention TREMOR), impaired force and rhythm of diadochokinesis (rapidly alternating movements), and GAIT ATAXIA. (From Adams et al., Principles of Neurology, 6th ed, p90)

关联

项与小脑共济失调相关的药物

Levacetylleucine

N-乙酰-L-亮氨酸

靶点

calcium channel

作用机制

calcium channel调节剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2024-09-24

Plastoquinonyl decyl triphenylphosphonium

靶点

50S subunit x Electron transport chain complex proteins

作用机制

50S subunit抑制剂 [+1]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

俄罗斯

首次获批日期2012-07-01

Luminol

鲁米诺

靶点-

作用机制

免疫调节剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段批准上市

首次获批国家/地区

匈牙利

首次获批日期2011-10-14

412

项与小脑共济失调相关的临床试验

NCT06366230

/ Not yet recruiting临床1/2期IIT

Adding Urea to the Final Dialysis Fluid in Order to Prevent Dialysis Disequilibrium in Patients Who Need Aggressive Dialysis for Electrolyte Abnormalities

At times patients with advanced renal failure present with severe hyperkalemia or acidosis and very high serum blood urea nitrogen (BUN) concentrations. These patients cannot be dialyzed aggressively as the lowering of serum BUN may results in disequilibrium syndrome but on the other hand they need aggressive dialysis in order to lower their serum potassium or fix their severe acidosis. If one is able to add urea to the dialysis fluid, one can prevent the rapid lowering of serum BUN and osmolality at the same time as doing aggressive dialysis to lower serum potassium and/or fix the metabolic acidosis.

开始日期2025-07-01

申办/合作机构

The University of California, San Francisco

NCT06177626

/ Not yet recruitingN/AIIT

Use of a New Smartphone Application, IBlink, to Determine Changes in Eyeblink Conditioning from Home Training in Individuals with Spinocerebellar Ataxias

Spinocerebellar ataxias are a group of disorders that cause severe disability and can be fatal. There are currently no known disease-modifying treatments available for use, and there is a critical need to find treatments that slow disease progression and allow affected individuals to live more functional lives. Aerobic training show promise as a treatment for these diseases, but it is unclear if training induces neuroplastic changes within the damaged cerebellum to enhance motor learning, or if improvements are primarily caused by changes in leg strength, fatigue, and endurance. It is crucial to understand how the training impacts the brain, and particularly the cerebellum, in order to determine the most effective training regimen.
To examine the impact of aerobic exercise on the brain, we propose using eyeblink conditioning, a form of motor learning that is dependent on the cerebellum. We will utilize BlinkLab, a newly developed smartphone application, that overcomes the typical barriers of testing eyeblink conditioning by allowing in-home assessments without the need for expensive equipment. We hypothesize that: 1) individuals with spinocerebellar ataxia will have impaired eyeblink conditioning, and 2) aerobic exercise, but not balance training, will improve eyeblink conditioning in this population. If these hypotheses are found to be true, it would further support that aerobic exercise is able to enhance motor learning in individuals with cerebellar damage.
In AIM 1, we will test eyeblink conditioning in individuals with ataxias and follow them over time to see if eyeblink conditioning might be a biomarker for cerebellar ataxia disease progression. We will then use these preliminary results to devise a larger study to further validate eyeblink conditioning as a biomarker for ataxia disease progression. In AIM 2, we will determine the impact of training on eyeblink conditioning. We expect that aerobic training, but not balance training, will enhance eyeblink conditioning in spinocerebellar ataxia. Finally, in AIM 3, we will explore the use of eyeblink conditioning as a biomarker of neuroplasticity.

开始日期2025-07-01

申办/合作机构

Columbia University

NCT03378414

/ Not yet recruiting临床2期

A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia

The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC therapy in Spinocerebellar Ataxia.

开始日期2025-06-30

申办/合作机构

圣释（北京）生物工程有限公司

100 项与小脑共济失调相关的临床结果

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100 项与小脑共济失调相关的转化医学

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0 项与小脑共济失调相关的专利（医药）

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64,953

项与小脑共济失调相关的文献（医药）

2026-01-01·Neural Regeneration Research

GEMIN5 and neurodevelopmental diseases: From functional insights to disease perception

Article

作者: Francisco-Velilla, Rosario ; Martinez-Salas, Encarnacion

GEMIN5 is a predominantly cytoplasmic multifunctional protein, known to be involved in recognizing snRNAs through its WD40 repeats domain placed at the N-terminus. A dimerization domain in the middle region acts as a hub for protein–protein interaction, while a non-canonical RNA-binding site is placed towards the C-terminus. The singular organization of structural domains present in GEMIN5 enables this protein to perform multiple functions through its ability to interact with distinct partners, both RNAs and proteins. This protein exerts a different role in translation regulation depending on its physiological state, such that while GEMIN5 down-regulates global RNA translation, the C-terminal half of the protein promotes translation of its mRNA. Additionally, GEMIN5 is responsible for the preferential partitioning of mRNAs into polysomes. Besides selective translation, GEMIN5 forms part of distinct ribonucleoprotein complexes, reflecting the dynamic organization of macromolecular complexes in response to internal and external signals. In accordance with its contribution to fundamental cellular processes, recent reports described clinical loss of function mutants suggesting that GEMIN5 deficiency is detrimental to cell growth and survival. Remarkably, patients carrying GEMIN5 biallelic variants suffer from neurodevelopmental delay, hypotonia, and cerebellar ataxia. Molecular analyses of individual variants, which are defective in protein dimerization, display decreased levels of ribosome association, reinforcing the involvement of the protein in translation regulation. Importantly, the number of clinical variants and the phenotypic spectrum associated with GEMIN5 disorders is increasing as the knowledge of the protein functions and the pathways linked to its activity augments. Here we discuss relevant advances concerning the functional and structural features of GEMIN5 and its separate domains in RNA-binding, protein interactome, and translation regulation, and how these data can help to understand the involvement of protein malfunction in clinical variants found in patients developing neurodevelopmental disorders.

2025-12-31·Cell Adhesion & Migration

Brief biology and pathophysiology of Tekt bundles

Review

作者: Liu, Wei ; Ni, Bing ; Yu, Zhongying ; Cheng, Yimei ; He, Wenjuan ; Wang, Xiao ; Miao, Hongming ; Zhang, Qinghua ; Yin, Jun ; Liu, Min ; Bai, Jialian

Tektins, a family of microtubule-stabilizing proteins, are critical for cilia and flagella assembly in mammals. They maintain doublet microtubule stability and ciliary/flagellar motility. Loss of Tekt1-5 causes microtubule instability, impaired motility, and diseases like infertility, retinal degeneration, Mainzer-Saldino syndrome, and diabetic nephropathy. Pathophysiological stimuli regulate Tektin expression through transcriptional, posttranscriptional, translational, and posttranslational modifications. This review summarizes the latest findings on Tektin functions and their role in diseases.

2025-12-31·Case Reports in Plastic Surgery and Hand Surgery

Levator aponeurosis advancement with partial orbicularis oculi muscle resection for the treatment of Marin Amat syndrome with aponeurotic ptosis: two Case reports

Article

作者: Yokoyama, Sho ; Tanabe, Yosihiko ; Kurihara, Satomi ; Osawa, Ikubun ; Noguchi, Kaito

Marin Amat syndrome is a phenomenon in which eyelids close upon opening of the mouth during the recovery phase after facial nerve paralysis. In this report, we present two surgically treated cases of Marin Amat syndrome with aponeurotic ptosis. Case 1: A 66-year-old man had developed left Bell's palsy a year prior to presentation and underwent rehabilitation at the Neurology Department of Japan Community Healthcare Organization Chukyo Hospital. He was subsequently referred to the Ophthalmology Department for left eyelid ptosis. Case 2: A 75-year-old man developed left Bell's palsy more than 10 years prior to presentation and was referred to the Ophthalmology Department for left eyelid ptosis. Both patients had Marin Amat syndrome with aponeurotic ptosis. Levator aponeurosis advancement surgery and partial orbicularis oculi muscle resection were performed on the affected eyes. Both patients showed favorable postoperative outcomes. Simultaneous surgery involving levator aponeurosis advancement and partial orbicularis oculi muscle resection is effective for treating Marin Amat syndrome with aponeurotic ptosis.

818

项与小脑共济失调相关的新闻（医药）

2025-04-30

·ioncology

仁心无国界丨林岩松教授美国MGH访学：晚期甲状腺癌靶向治疗观点抢先看

点击上方蓝字关注我们日前，北京协和医院林岩松教授前往美国麻省总医院（MGH）进行为期三天的访学，旨在加强双方在甲状腺癌治疗领域的合作与交流。在此次访学中，林教授与MGH甲状腺癌领域的专家进行了深入交流。《肿瘤瞭望》在参访过程中特别采访了林岩松教授与MGH头颈部肿瘤医学主任Lori Wirth教授。在此次访谈中，两位专家立足于中美两国就晚期甲状腺癌的现状、诊疗模式及规范，患者管理等方面交换了观点，同时，对《2024年美国甲状腺癌协会（ATA）成人分化型甲状腺癌患者管理指南（征求意见稿）》中靶向治疗部分的更新进行了深入探讨，并展望了靶向治疗单药及靶向免疫联合方案在分化型甲状腺癌（DTC）和未分化型甲状腺癌（ATC）的应用前景和探索方向。此次参访为中美两国在甲状腺癌治疗领域的学术交流搭建了重要平台，也将为两国晚期甲状腺癌患者的诊疗带来新的思考。以下为林教授观点抢先看，更多外方观点及详细讨论内容，将在下期栏目中特别呈现，敬请期待。林岩松教授与Lori Wirth教授01肿瘤瞭望：请简要分享当前中国在晚期甲状腺癌诊疗及患者管理方面的发展现状及特点。林岩松教授北京协和医院中国甲状腺癌的疾病负担较重，发病率较高，中国每年新增甲状腺癌病例数占全球新增病例数的一半以上，五年生存率（OS）不足93%，与美国超过98%的5年OS率相比仍有差距[1,2]。这一差距凸显了综合管理的重要性和紧迫性。近年来，中国多个学协会发布了多项指南和共识，对甲状腺癌的规范化管理起到了重要作用。然而，中国仍面临医疗资源分布不均、多学科团队协作不足（尤其是偏远地区）等挑战，中国甲状腺疾病领域专家仍需为之努力，以提升整体治疗水平。02肿瘤瞭望：请您谈谈中国在放射性碘难治性分化型甲状腺癌（RAIR-DTC）的多学科诊疗模式是怎样的？会如何决策后续治疗方案？林岩松教授北京协和医院在中国，核医学科专家是甲状腺癌患者放射性碘（RAI）治疗的决策者，同时也负责识别放射性碘难治性分化型甲状腺癌（RAIR-DTC）。RAIR-DTC是中国晚期甲状腺癌的主要患者群体，其治疗的复杂性要求多学科团队（MDT）的紧密协作；在MDT的模式下，对于局部复发的RAIR-DTC患者，手术治疗是首选方案；而对于广泛转移的RR-DTC患者，则以靶向治疗药物（如酪氨酸激酶抑制剂，TKI）为主，进行系统治疗。03肿瘤瞭望：请您谈谈中国在指南或规范上对于晚期甲状腺癌靶向治疗临床应用国内外指南对中国临床实践的影响？林岩松教授北京协和医院2020年，中国国家药品监督管理局（NMPA）批准仑伐替尼（DTC适应症）上市，并陆续批准了RET抑制剂等靶向药物，为患者提供了多样化的治疗选择。然而由于药物可及性等原因，目前中美的治疗存在差异；例如卡博替尼是美国的标准二线治疗方案[3]，但在中国尚未获批，导致中国在二线治疗方面缺乏统一标准，更多依赖于个体化的临床实践。为规范临床实践，中国多个学协会积极制定了多项指南和共识，为RAIR-DTC、ATC和髓样甲状腺癌（MTC）等提供了详细诊疗建议，推动了中国晚期甲状腺癌治疗的规范化。2024年6月，中国专家撰写并发布了《放射性碘难治性分化型甲状腺诊治管理指南（2024版）》[4]，其中与《2024年美国甲状腺癌协会（ATA）成人分化型甲状腺癌患者管理指南（征求意见稿）》中许多观点不谋而合，我们高度关注并期待新版ATA指南的正式发布。04肿瘤瞭望：以仑伐替尼为代表的多靶点TKI已经成为RAIR-DTC的标准治疗方案，并获得多部国内外指南一线推荐。请您谈谈仑伐替尼在中国RAIR-DTC的临床应用价值及前景？林岩松教授北京协和医院在中国，基于SELECT研究[5]、308研究[6]以及仑伐替尼的事后分析数据提示，仑伐替尼在延长患者无进展生存期（PFS）和65岁以上患者的总生存期（OS）方面表现出显著优势[7]。这些研究结果进一步巩固了仑伐替尼在中国指南中的优先推荐地位，使其成为RAIR-DTC的标准治疗方案。尽管中国已有多款靶向药物获批，如RET抑制剂，但由于突变率较低、价格等因素，仑伐替尼仍是临床实践中的首选。05肿瘤瞭望：国内外指南都强调，对于晚期或难治性甲状腺癌，建议积极探索新的治疗策略，比如靶免联合方案。请您谈谈靶免联合治疗的应用前景及临床获益。林岩松教授北京协和医院靶免联合治疗可能成为未来的治疗趋势和选择。TKI单药虽然在RAIR-DTC中取得了良好疗效，耐药的发生不可避免。但以仑伐替尼为代表的靶向药物通过抑制VEGFR通路改善肿瘤微环境，从而增强免疫治疗的效果，可与免疫治疗发挥协同作用，在已有临床研究中，仑伐替尼联合免疫治疗在未分化型甲状腺癌的ORR超过50%，在甲状腺低分化癌的ORR超过70%，靶向免疫联合治疗的初步结果令人惊喜[8]。在中国，靶免联合方案也会作为二线治疗的一种选择。结束语在本次中美甲状腺癌治疗领域的深度交流中，林岩松教授分享了中国专家在甲状腺癌规范化管理的治疗经验与进展。下一期Lori Wirth教授将与林教授一起就中美两国在甲状腺癌精准治疗、靶向药物应用及多学科协作等方面的前沿理念进行深度交流，相信这些观点将为两国晚期甲状腺癌诊疗带来更多思路，为临床实践提供有益参考。敬请锁定《仁心无国界》栏目，期待更多精彩内容，让仁心跨越国界，为患者点亮生命之光。参考文献上下滑动查看更多内容[1] Zeng H, Zheng R, Sun K, et al. Cancer survival statistics in China 2019-2021: a multicenter, population-based study. J Natl Cancer Cent. 2024;4(3):203-213. Published 2024 Jun 22. doi:10.1016/j.jncc.2024.06.005[2] Miller KD, Nogueira L, Devasia T, et al. Cancer treatment and survivorship statistics, 2022. CA Cancer J Clin. 2022;72(5):409-436. doi:10.3322/caac.21731[3] National Comprehensive Cancer Network®. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Thyroid Carcinoma Version 5.2024. Available at [2025.03.08] : https://www.nccn.org/professionals/physician_gls/pdf/thyroid.pdf[4] 中国临床肿瘤学会核医学专家委员会,中国临床肿瘤学会甲状腺癌专家委员会,中华医学会核医学分会,等. 放射性碘难治性分化型甲状腺癌诊治管理指南(2024版)[J]. 中华核医学与分子影像杂志,2024,44(6):359-372. DOI:10.3760/cma.j.cn321828-20240125-00034.[5] Schlumberger M, Tahara M, Wirth LJ, et al. Lenvatinib versus placebo in radioiodine-refractory thyroid cancer. N Engl J Med. 2015;372(7):621-630. doi:10.1056/NEJMoa1406470[6] Zheng X, Xu Z, Ji Q, et al. A Randomized, Phase III Study of Lenvatinib in Chinese Patients with Radioiodine-Refractory Differentiated Thyroid Cancer. Clin Cancer Res. 2021;27(20):5502-5509. doi:10.1158/1078-0432.CCR-21-0761[7] Brose MS, Worden FP, Newbold KL, Guo M, Hurria A. Effect of Age on the Efficacy and Safety of Lenvatinib in Radioiodine-Refractory Differentiated Thyroid Cancer in the Phase III SELECT Trial. J Clin Oncol. 2017;35(23):2692-2699. doi:10.1200/JCO.2016.71.6472[8] Dierks C, Seufert J, et al. 2022 ESMO 1646MO-Phase II ATLEP trial: Final results for lenvatinib/pembrolizumab in metastasized anaplastic and poorly differentiated thyroid carcinoma . Annals of Oncology (2022) 33 (suppl_7): S750-S757. 10.1016/annonc/annonc1077.注：本文根据英文采访编译整理，采访原视频下期呈现，敬请期待（来源：《肿瘤瞭望》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

放射疗法

2025-04-30

·生物制药小编

13条研发管线，斩获6亿美元投资

近日Biohaven公司与Oberland Capital达成一项非稀释性融资协议，公司将获得来自Oberland Capital的总价高达6亿美元的投资。据协议，该笔投资将分为三批，其中首批投资（2.5亿美元）将在2025年4月30日或之前到账，第二批（1.5亿美元）投资在FDA对troriluzole的做出批准决定后，由公司决定是否兑现，剩下2亿美元将在双方达成一致的情况下，用于支持公司进行战略收购等支出。作为交换，Oberland Capita有权获得troriluzole的监管里程碑付款，这些付款将按季度支付，直至2030年12月31日。另外，Biohaven还将支付Oberland Capita未来最多10年troriluzole的销售分成（个位数比例）。被收购后原地拆分Biohaven最早成立于2013年，是一家专注于神经以及精神疾病疗法开发的Biotech公司。2020年，公司推出了全球首个治疗及预防偏头痛药物Nurtec ODT（口服CGRP受体拮抗剂），上市之后Nurtec ODT销售表现强劲，第一个完整销售年（2021年）便卖出4.63亿美元，成为偏头痛类药物中排名第一的处方药。辉瑞看重了公司其CGRP受体拮抗剂在偏头痛领域的市场前景，辉瑞在2021年11月与Biohaven达成合作，以5亿美元首付款获得Nurtec以及另外一款CGRP受体拮抗剂Zavegepant（鼻喷）的部分商业化权益。2022年5月，辉瑞直接宣布以116亿美元现金收购了Biohaven所有的CGRP管线，而Biohaven管线中非CGRP项目则被保留下来。收购完成后，原Biohaven成为辉瑞的全资子公司，而同时，基于剩下的非CGRP管线，有原地拆分出一家仍以Biohaven名称的新公司，并且为独立上市公司运营。13条研发管线拆分之后，新的Biohaven现在拥有有13条在研管线，其中最快的为troriluzole（谷氨酸调节剂）的上市申请已经获得了FDA受理并授予了优先审评，用于治疗脊髓小脑性共济失调（SCA）。其中BHV-7000（钾离子通道激活剂）和BHV-2000（肌生长抑制素阻断剂）进入到临床Ⅲ期。不过，BHV-7000今年3月报告了坏消息，在针对I型双相障碍狂躁症发作的II/III期临床试验中，相比对照组，BHV-7000在Young Mania评定量表评分上未能达成显著差异。目前，公司比较惹人注目的是其蛋白降解剂管线。其建立了一种胞外蛋白降解剂技术平台MoDE，使小分子结合细胞外靶蛋白，使其通过肝脏从体内排出。MoDE是一种双特异性分子，一端结合肝细胞上的ASGPR，另一端结合胞外目标蛋白。BHV-1400（Gd-IgA1降解剂）、BHV-1300（IgG降解剂）两款蛋白降解剂在都在临床中显示出色的降解能力。据该今年1月公布的BHV-1400（治疗IgA肾病）Ⅰ期临床中，给药4小时内，最低剂量BHV-1400（125mg）便可将Gd-IgA1水平平均降低60%，8小时内最大降幅超过70%。且单次给药后，BHV-1400对Gd-IgA1的降解效果能持续数天。据Biohaven称，BHV-1400的降解效果是目前针对Gd-IgA1药物中前所未有的。基于此数据，Biohaven计划将其直接推上III期临床。今年3月公布的BHV-1300 Ⅰ期临床数据显示，给药4周后，BHV-1300（1000mg）可将致病性IgG水平平均降低80%，且在单次给药后数小时内即可快速、持续降低IgG水平。Biohaven同步推进这些管线的临床开发需要消耗大量的资金，根据公司2024年财报，截至2024年12月31日，公司现金、现金等价物、有价证券以及受限现金总计约4.89亿美元。参考出处https://www.fiercebiotech.com/biotech/biohaven-after-setbacks-sees-shares-jump-600m-oberland-capital-funding-agreementhttps://ir.biohaven.com/news-releases/news-release-details/biohaven-announces-investment-600-million-oberland-capitalhttps://reportify.cn/filings/1094181338036178944

并购优先审批临床3期申请上市

2025-04-29

·Pharmaceutical Technology

Biohaven secures $600m financing as it awaits FDA prodrug decision

Biohaven has secured funding to support its potential drug launch and pipeline growth. Credit: Olena Malik via Getty Images. Biohaven has secured a funding deal worth up to $600m from Oberland Capital Management, providing the biotech with non-dilutive capital as it prepares for a potential US launch of troriluzole for spinocerebellar ataxia (SCA). The financing comes in the form of a note purchase agreement, allowing Oberland to acquire up to $600m of Biohaven’s senior secured notes in multiple tranches. The first tranche of $250m is scheduled to be funded by 30 April, according to the company statement issued on 28 April. The second tranche – worth $150m – is contingent on US Food and Drug Administration (FDA) approval of troriluzole and subject to additional unspecified conditions. A third tranche of $200m may be made available for strategic acquisitions and related expenses. Oberland Capital will be eligible for a regulatory approval milestone payment, payable quarterly through the end of 2030. Shares in Biohaven closed 9.6% higher at $21.74 on 28 April, compared to $19.84 at market open. The deal comes as Biohaven awaits a regulatory decision from the FDA on troriluzole. The candidate is a prodrug of riluzole, which is already approved for amyotrophic lateral sclerosis (ALS). Troriluzole is being evaluated for SCA , a rare neurodegenerative disorder with limited treatment options, characterised by a loss of coordination and balance. The FDA granted the application priority review in February 2025, with a final decision expected in Q3 2025. The company is positioning the new funding to accelerate commercial planning and advance its broader pipeline, which includes CNS-targeting therapies. 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 Biohaven’s chief financial officer Matt Buten said: “This agreement with Oberland Capital provides us with the support and flexibility to continue advancing our pipeline as we make launch preparations in parallel, and we are excited to accelerate our planning efforts in earnest.” If it wins approval, troriluzole would represent Biohaven’s first greenlit product since it spun out from its migraine business following a $11.6bn acquisition from Pfizer back in 2022. However, Biohaven’s path to approval for troriluzole has not been straightforward. The drug failed to meet its primary endpoint in a Phase III trial (NCT03701399) conducted in 2022. Despite the setback, Biohaven shared subsequent analyses revealing benefits for patients with a certain subset of the disease and showed a slowed disease progression. The FDA initially refused to review the application but later accepted a revised submission following the release of this ad hoc data. Troriluzole’s prospects in Europe are more uncertain. Earlier in April, the European Medicines Agency (EMA) questioned whether the drug’s efficacy meaningfully differed from that of riluzole and challenged its classification as a new active substance. According to the EMA, Biohaven said it would generate additional data before submitting a new application. The new Biohaven entity was formed after the divestiture of its migraine assets to Pfizer in 2022. That deal included the commercial rights to Nurtec ODT (rimegepant), which generated $1.26bn in global sales in 2024, as per Pfizer’s financials. In January 2025, Pfizer agreed to pay $59.7m to settle allegations related to illegal payments made by the former Biohaven entity to healthcare providers before its acquisition. The US Department of Justice alleged that Biohaven paid kickbacks to physicians to boost prescriptions of Nurtec, resulting in false claims submitted to Medicare and other federal healthcare programmes between March 2020 and September 2022.

并购临床3期优先审批申请上市上市批准