Observational study on women with Cystic Fibrosis treated with CFTR modulators during pregnancy and postpartum and their children. Registration on maternal health parameters and effects of CFTR-modulators in the newborn infant as well as effects of exposure through mother's own milk.

开始日期2024-03-15

申办/合作机构

Region Stockholm

[+1]

NCT06154447

/ Recruiting临床1期

A Phase 1, Study of VX-828 in Healthy Subjects and in Subjects With Cystic Fibrosis

The purpose of the study is to evaluate safety, tolerability, and pharmacokinetics of VX-828 and VX-828 in triple combination (TC) with Tezacaftor (TEZ)/ VX-118 or TEZ/ deutivacaftor (D-IVA) in healthy participants and VX-828 in combination with D-IVA with or without TEZ in participants with cystic fibrosis (CF).

开始日期2023-12-12

申办/合作机构

Vertex Pharmaceuticals, Inc.

NCT05822102

/ Unknown statusN/AIIT

Simultaneous quanTification of Elexacaftor/tezAcaftor/Ivacaftor Via Reverse Phase High Performance liquiD chromatographY

The purpose of this study is to support the clinical validation of a new assay to measure the levels of ivacaftor, tezacaftor, and elexacaftor (the components of Trikafta) in the bloodstream in order to achieve greater understanding of the effectiveness of this medication in all people with cystic fibrosis. Blood will be drawn at 3.0, 4.5, and 6.0 hours after taking the medication in the morning.

开始日期2023-02-09

申办/合作机构

National Jewish Health, Inc.

100 项与替扎卡托相关的临床结果

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100 项与替扎卡托相关的转化医学

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100 项与替扎卡托相关的专利（医药）

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1,042

项与替扎卡托相关的文献（医药）

2025-12-31·European Clinical Respiratory Journal

The complexities of elexacaftor/tezacaftor/ivacaftor therapeutic drug monitoring in a person with cystic fibrosis and Mycobacterium abscessus pulmonary disease

Article

作者: Riddles, Timothy ; Evans, Ieuan E S ; Matson, Angela G ; Johnson, Ellie ; Mou, Claire Y ; Reid, David W ; Henderson, Daniel J ; Swenson, Rebecca ; Herd, Karen M ; McWhinney, Brett ; Burke, Andrew

Therapeutic drug monitoring (TDM) of elexacaftor/tezacaftor/ivacaftor (ETI) remains challenging due to a lack of clarity around the parameters that govern ETI plasma concentrations, whilst the use of concomitant CYP3A inducers rifabutin and rifampicin is not recommended. We present the complexities of TDM for ETI performed in a person with cystic fibrosis and refractory Mycobacterium abscessus pulmonary disease. Utilising National Association of Testing Authorities (NATA) accredited assays and target considerations published by the Therapeutic Goods Administration (TGA), Australia, ETI plasma concentration variability was monitored over the course of an acute admission with added complexity from an antibiotic regimen including rifabutin, a moderate cytochrome P450 3A (CYP3A) inducer, and clofazimine, a mild CYP3A inhibitor. This case highlights the challenges surrounding ETI TDM in the context of acute severe illness, malnutrition, chronic infection, and drug-to-drug interactions. The marked clinical improvement seen, alongside sustained ETI plasma concentrations and suppressed sweat chloride levels on serial testing, provided reassurance of the use of ETI and rifabutin concomitantly in this case, and highlights the potential utility of TDM in helping guide clinical practice. Though a current barrier to the application of TDM includes ETI only being available as a fixed dose combination.

2025-12-01·LUNG

The Role of Triple CFTR Modulator Therapy in Reducing Systemic Inflammation in Cystic Fibrosis

Article

作者: Punter, Rosa Mar Gómez ; Alonso, Esther Ferreira ; Ancochea, Julio ; Madrid-Carbajal, Claudia Janeth ; Moreno, Rosa María Girón ; Ruiz, Josué David Hernández ; García, Belén Prieto ; Peláez, Adrián ; Clemente, Marta García ; García, Marta Solís ; Bachiller, Jose María Eiros

PURPOSE:

Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTR gene, leading to multisystemic complications, particularly in the lungs. CFTR dysfunction results in altered ion transport, chronic inflammation, and progressive lung damage. The triple therapy elexacaftor/tezacaftor/ivacaftor (ETI) has demonstrated significant improvements in pulmonary function and quality of life. This study aimed to evaluate the anti-inflammatory effects of ETI by analysing systemic cytokine profiles over 12 months.

METHODS:

A prospective study included 32 CF patients ≥ 18 years with at least one delF508 mutation, undergoing ETI therapy. Clinical stability was ensured prior to therapy initiation. Demographic data, BMI (Body Mass Index), FEV1% (Forced expiratory Volume in the first second), VR/TLC (residual volume/total lung capacity) and sweat chloride concentrations were recorded at baseline, 6 months and 12 months. Inflammatory markers, including fibrinogen, C-reactive protein (CRP), and a panel of 8 cytokines, were measured using multiplex bead-based immunoassays and electrochemiluminescence. Longitudinal changes were analysed using mixed-effects models and statistical tests, with significance set at p < 0.05.

RESULTS:

During a 12-month follow-up, the neutrophils number and proinflammatory biomarkers analyzed, fibrinogen, CRP, GM-CSF, IFN- γ, IL-1 α, IL-1 β, IL-8 (CXCL8), IL-12p70, IL-17A (CTLA-8), and TNF-α, significantly decreased, while eosinophils remained stable. Mixed-effects models confirmed the significant association of inflammatory biomarkers with FEV1, BMI, sweat chloride levels, and VR/TLC highlighting the role of inflammation in the progression of CF.

CONCLUSIONS:

ETI demonstrated marked anti-inflammatory effects in CF patients, reducing systemic inflammation and improving clinical parameters.

2025-12-01·LUNG

Lung Clearance Index Improves in People with Cystic Fibrosis not Achieving a Clinical Important Difference in Forced Expiratory Volume in One Second After Elexacaftor/Tezacaftor/Ivacaftor Therapy

Article

作者: Lanfranchi, Chiara ; Alicandro, Gianfranco ; Mariani, Alessandra ; Bellante, Federica ; Rosazza, Chiara ; Biffi, Arianna ; Zazzeron, Laura ; Gramegna, Andrea ; Daccò, Valeria ; Blasi, Francesco

PURPOSE:

In people with cystic fibrosis (pwCF), elexacaftor/tezacaftor/ivacaftor (ETI) therapy is associated with an average improvement in FEV₁ of 10-14%. However, a subset of individuals fails to achieve a clinically meaningful increase in spirometric indicators. In this study, we aimed to assess whether the lung clearance index (LCI_2.5), a more sensitive indicator of lung involvement, improves following ETI initiation in this population.

METHODS:

We conducted a prospective observational study in a specialized CF center in Italy. PwCF performed a spirometry and a multiple breath nitrogen washout test the day they initiated ETI therapy and after 6 and 12 months. They were grouped according to the 12-month change in FEV₁ into two groups: Individuals who experienced a change in FEV₁ ≥ a minimal clinically important difference (MCID) of 3% and those who did not. Mean changes in LCI_2.5 were estimated using generalized estimating equations.

RESULTS:

The study included 129 pwCF who initiated ETI at our center (Age Range: 12-36 years). In 20 subjects (15.5%), the FEV₁ change was < MCID. These individuals had better baseline pulmonary function than those with FEV₁ changes ≥ MCID (Median FEV₁: 102.5 vs 87.0%), with the majority (90%) having FEV₁ values ≥ 90%. Mean changes in LCI_2.5 at 12-month follow-up visit were - 1.44 units (95% CI: - 2.12; - 0.75) in individuals with changes in FEV₁ < MCID and - 2.64 units (95% CI: -3.05; -2.23) in those with values ≥ MCID.

CONCLUSION:

LCI_2.5 is a useful measure to monitor the effectiveness of ETI in pwCF with normal spirometry and limited FEV₁ change following treatment initiation.

208

项与替扎卡托相关的新闻（医药）

2025-08-28

·药明康德

致敬时代 | 打破死亡“诅咒”，他们用数十年改写这类患者的命运

编者按：2025年，药明康德迎来创立25周年的重要里程碑。值此契机，我们向所有与我们共同书写产业变革篇章的科学家、医药人和投资者致以衷心感谢与诚挚敬意，也特别推出“致敬时代”系列，回顾全球同仁如何借助科学与合作的力量，不断拓展治疗边界、改善患者命运。四分之一个世纪的坚守，只为加速每一款新药的诞生。下一个25年，我们将继续心怀感恩与敬畏，依托独特的CRDMO模式，与全球伙伴携手同行，共赴健康未来。揭开被“诅咒”的真相数个世纪以前，在欧洲的部分地区流传着这样一个说法：“当亲吻婴儿额头能尝到咸味时，那么他就受到了诅咒，很快就会死去。”在当时，这种诅咒之说具有很强的信服力，因为婴儿除了咸咸的皮肤，还会持续咳嗽，体重难以增长并最终死亡。直到20世纪，诅咒之说才迎来了全新的科学解释。1938年，美国病理学家多萝西·安德森（Dorothy Andersen）博士在解剖营养不良儿童尸体的过程中观察到，一些患儿存在胰管扩张呈囊状和广泛纤维化现象。她将此描述为“胰腺囊性纤维化”，这也是囊性纤维化（cystic fibrosis，CF）一词首次出现在科学界的视野中。 1949年，儿科病理学家保罗·迪桑特·阿格涅斯（Paul di Sant’Agnese）博士注意到，囊性纤维化患者汗液中的盐分明显比正常人的更高。基于这一发现，阿格涅斯医生发明了用汗液诊断囊性纤维化的新方法。进入20世纪80年代，在加州大学圣地亚哥分校担任研究员的保罗·昆顿（Paul Quinton）教授揭开了“咸吻”的来源。他发现，囊性纤维化患者汗腺上皮细胞的氯离子通道存在异常，不仅影响汗液中的氯离子重吸收、导致了“咸吻”，还会破坏器官细胞的盐分与水分平衡，造成黏液聚集。图片来源：123RF 随着基因层面研究的发展，氯离子通道失常的深层原因也有了答案。1989年，徐立之教授、弗朗西斯·柯林斯（Francis Collins）教授、杰克·赖尔登（Jack Riordan）教授组成的联合团队找到并命名了真正影响囊性纤维化发生的基因——囊性纤维化跨膜转导调节因子（CFTR）。研究显示，CFTR基因编码的蛋白质能在上皮细胞表面形成离子通道。该通道通过调控氯离子和碳酸氢根离子的跨膜转运，维持黏液中的盐水平衡。当CFTR基因突变，黏液会异常黏稠，从而阻塞气道和导管。这些科学家还发现，囊性纤维化最常见的CFTR F508del突变，仅仅因为一个苯丙氨酸的缺失就使得CFTR蛋白无法正确折叠，难以抵达细胞表面。即使有极少蛋白到达细胞膜，也处于失效状态，无法正常控制跨膜通道。近25载求索，重塑生命篇章 CFTR基因的发现彻底改变了囊性纤维化的治疗格局。科学家第一次可以试图从根源修复缺陷，而不是简单地应对症状。最初，囊性纤维化基金会与生物技术公司Aurora Biosciences进行了合作，寻找可能改善缺陷CFTR蛋白功能的化合物。2001年，Vertex Pharmaceuticals收购了该公司，并继续推进囊性纤维化的研究。那一年，33岁的弗雷德里克·范古尔（Fredrick Van Goor）博士在Vertex Pharmaceuticals开始了全新征程，他的上级提出了一个问题：“你知道导致囊性纤维化的遗传因素吗？就是那个致命的肺部疾病。” 范古尔博士当时并不了解这种疾病，因此回答：“不知道。”然而，后续的交谈让他心里产生了一个大胆的想法，他想要给这种疾病找到治愈方法。他告诉自己，“我可以回答‘我知道’，之后我会把有关它的问题弄清楚。” 在此后的20多年里，他一直在履行着自己的承诺。范古尔博士成为了Vertex Pharmaceuticals囊性纤维化研究项目的核心成员。为了更好地破解这一疾病，他经常奔走在实验室与医院间，收集数据的同时与患者进行交谈。在数百次的谈话中，他对一名患者的回答记忆犹新，当时这名患者将自己描述身处在一艘沉没的轮船上，而救生艇都已经离开。范古尔博士要做的，就是重新为他们打造一艘拯救生命的“救生艇”。范古尔博士和合作者提出了两种治疗分子思路：一种是发挥校正剂（corrector）的效果，引导突变蛋白前往细胞膜；另一种则是增效剂（potentiator），可以帮助蛋白到达位置后稳定发挥作用。接下来，研究团队筛选出一个靶向CFTR蛋白上G551D突变位点的分子VX-770。该位点突变时，CFTR蛋白作为离子通道的打开速度会变慢，而VX-770可以作为增效剂帮助打开跨膜通道。在临床试验中，VX-770可以减少患者呼吸道感染的发作次数，并改善呼吸功能。2012年, FDA批准了首个针对囊性纤维化病因研发的药物ivacaftor（商品名Kalydeco，即VX-770）上市，用于治疗携带G551D突变的囊性纤维化患者。《新英格兰医学杂志》的一篇社论文章将ivacaftor描述为囊性纤维化领域的重要里程碑。尽管携带G551D突变的患者比例不大，但这一突破性结果证明了范古尔博士思路的正确性，囊性纤维化不再是一种无从下手的疾病。图片来源：123RF 有了Kalydeco的成功案例，研究团队开始尝试攻克更常见的F508del突变。F508del突变的难度在于，除了增效剂，研究团队还需要设计一种校正剂，帮助CFTR蛋白前往细胞膜。为此，他们研发了一种新药lumacaftor。Lumacaftor搭配ivacaftor使用时，可同时解决F508del突变的两种问题。2015年7月，囊性纤维化的第一种联合疗法Orkambi（ivacaftor/lumacaftor）获FDA批准上市，用于治疗6岁及以上拥有两个F508del突变拷贝的囊性纤维化患者。2018年2月，另一种校正剂tezacaftor与ivacaftor组合的药物Symdeko获得FDA批准，用于治疗12岁以上携带两个F508del突变拷贝的囊性纤维化患者。 Orkambi与Symdeko的诞生不是终点，范古尔博士团队在tezacaftor基础上增加了第二种校正剂elexacaftor，三种药物分子组合而成的Trikafta（elexacaftor/tezacaftor/ivacaftor）诞生了。两种校正剂作用于CFTR蛋白的不同部分，产生协同效应。相比于早期获批的药物，这种组合能够惠及仅携带一个F508del突变的患者。FDA于2019年10月批准Trikafta用于治疗12岁以上至少携带一个F508del突变的囊性纤维化患者。 Trikafta的成功也让组合治疗的思路在囊性纤维化领域得到进一步发展。2024年底，囊性纤维化患者又迎来了一个好消息，Vertex Pharmaceuticals的新一代CFTR调节剂组合——Alyftrek（deutivacaftor/tezacaftor/vanzacaftor）获FDA批准上市，用于治疗6岁及以上、携带至少一个F508del突变或其他对这款新药有应答的CFTR基因突变的囊性纤维化患者。 20多年的坚持为众多囊性纤维化患者带来了治愈希望。因开发出了用于治疗囊性纤维化的革命性药物，范古尔博士与Vertex Pharmaceuticals的另外两名科学家保罗·内古列斯库（Paul Negulescu）博士、萨宾·哈迪达（Sabine Hadida）博士获得了2024年生命科学突破奖（Breakthrough Prize in Life Sciences）。 CRDMO平台赋能新疗法研发这些药物的出现显著提升了囊性纤维化患者的生活质量，他们不再依赖其他耗时的治疗方法，并摆脱了呼吸道感染的威胁，得以更加自由地呼吸。从研发之初到药物诞生，长达20多年的探索与创新彻底变革了囊性纤维化的治疗格局。如今，囊性纤维化的药物研发呈现百花齐放的态势。一些团队正尝试借助mRNA疗法进一步帮助尚未受益的囊性纤维化患者。例如，ReCode Therapeutics公司的吸入式mRNA疗法通过脂质纳米颗粒（LNP）载体将疗法递送到肺部正确位置，表达功能性CFTR蛋白以治疗囊性纤维化。这款RCT2100疗法于今年3月获得FDA授予治疗囊性纤维化的孤儿药资格，已经进入临床开发阶段。此外，Arcturus Therapeutics公司开发的吸入式mRNA疗法ARCT-032已进入2期临床试验阶段，该疗法利用Arcturus的LUNAR脂质介导雾化平台递送CFTR mRNA抵达肺部，旨在表达功能性CFTR蛋白并减轻导致进行性肺部疾病的下游影响。与此同时，多种其他疗法也蕴育了治疗囊性纤维化的潜力，相关临床研究正在进行。在反义寡核苷酸（ASO）疗法领域，SpliSense公司在研的ASO疗法SPL84旨在治疗CFTR基因中携带特定剪接突变的患者，目前2期临床试验正在开展。4D Molecular Therapeutics公司的候选基因疗法4D-710通过腺相关病毒载体A101，以气溶胶形式将疗法递送至肺部从而恢复CFTR表达，目前4D-710处于1/2期临床试验阶段，此前公布的中期数据展现出积极结果。此外，一些噬菌体疗法正在尝试治疗囊性纤维化患者的慢性肺铜绿假单胞菌感染。药明康德在成立至今的25年里，见证了重磅囊性纤维化疗法的诞生与发展，也携手全球合作伙伴，在这一领域贡献自己的力量。作为创新的赋能者，药明康德长期以来也在为此类疾病新疗法的开发提供各种服务，通过一体化、端到端的CRDMO业务模式，从早期药物发现和研究（R）、开发（D）到生产（M）都能为合作伙伴提供支持，助力更多突破性疗法造福病患。根据公开信息，在过去3年中，药明康德纤维化药物开发一体化平台助力全球合作伙伴向美国FDA/中国NMPA提交了超过40个纤维化药物相关的IND申请；药明康德测试业务平台的药性评价部也能为包括CFTR蛋白调节剂在内的各类药物的体内、体外药物代谢与药代动力学（DMPK）研究提供支持。与过去相比，囊性纤维化患者的生存状况如今得到了空前的改善，但这场抗争还没有结束，面对无法从已有疗法中受益的囊性纤维化患者，能做的还有很多。曾担任囊性纤维化基金会主席普雷斯顿·坎贝尔（Preston Campbell）博士对未来有着乐观的期待：“让我们永远心怀梦想，梦想我们的抗争之路终有尽头。直到有一天，我们可以说，我曾经是一名囊性纤维化患者。” 参考资料： [1] Life-Changing Cystic Fibrosis Treatment Wins $3-Million Breakthrough Prize. Retrieved August 21, 2025 from https://www.scientificamerican.com/article/life-changing-cystic-fibrosis-treatment-wins-3-million-breakthrough-prize/ [2] Cystic fibrosis drugs target the malformed proteins at the root of the disease. Retrieved August 21, 2025 from https://www.nature.com/articles/d41586-020-02106-w [3] FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older Who Have at Least One F508del Mutation. Retrieved August 21, 2025 from https://www.businesswire.com/news/home/20191021005792/en [4] FDA approves new breakthrough therapy for cystic fibrosis. Retrieved August 21, 2025 from https://www.prnewswire.com/news-releases/fda-approves-new-breakthrough-therapy-for-cystic-fibrosis-300942263.html [5] Ratjen, F., Bell, S., Rowe, S. et al. Cystic fibrosis. Nat Rev Dis Primers 1, 15010 (2015). https://doi.org/10.1038/nrdp.2015.10 [6] ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis. Retrieved August 21, 2025 from https://recodetx.com/recode-therapeutics-receives-u-s-fda-orphan-drug-designation-for-rct2100-for-the-treatment-of-cystic-fibrosis/ [7] Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency. Retrieved August 21, 2025 from https://ir.arcturusrx.com/news-releases/news-release-details/arcturus-therapeutics-announces-initiation-dosing-phase-2 [8] 4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis. Retrieved August 21, 2025 from https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-announces-update-regulatory-interactions-and-development [9] A Drug Of Your Own. Retrieved August 21, 2025 from https://www.forbes.com/forbes/2011/0808/features-biotech-fred-van-goor-medicines-drug-your-own.html [10] Welsh MJ. Targeting the basic defect in cystic fibrosis. N Engl J Med. 2010 Nov 18;363(21):2056-7. doi: 10.1056/NEJMe1010123 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2025-08-06

·药事纵横

FDA当头棒喝！Vertex镇痛管线遭双重打击

8月4日，Vertex Pharmaceuticals（福泰制药）周一在盘后交易中股价一度重挫15%，因公司针对非阿片类镇痛布局连遭两项重大挫折：已获批用于急性疼痛的Journavx（suzetrigine）基本无缘“外周神经病理性疼痛”（PNP）这一广泛适应症；同时，下一代静脉用NaV1.8抑制剂VX-993在急性疼痛II期研究中未能击败安慰剂，单药开发就此终止。慢性疼痛扩张计划大幅收缩 Vertex首席执行官Reshma Kewalramani博士在当日投资者电话会上确认，在与美国食品药品监督管理局（FDA）完成II期结束后会议后，公司决定不再就“痛性腰骶神经根病”（LSR，俗称坐骨神经痛）开展新的III期试验，意味着Journavx短期内无法获得广泛PNP标签。 “虽然广泛的PNP标签仍是长期目标，但FDA目前看不到支持这一适应症的路径，” Kewalramani表示，“基于与FDA的明确共识，我们即将启动第二项糖尿病周围神经病变（DPN）III期研究，以确保DPN成为suzetrigine在PNP领域的首个获批适应症。” 去年，Journavx已获FDA授予DPN突破性疗法认定，首项III期研究已于去年启动。Vertex预计两项DPN III期试验将在2026年底前完成入组。 VX-993急性疼痛II期折戟 Vertex同时宣布，其新一代静脉注射用NaV1.8抑制剂VX-993在一项针对拇囊炎切除术后急性疼痛患者的II期研究中失败。与安慰剂相比，该候选药物未能带来具有统计学意义的疼痛缓解，因此，该制药商将停止其作为急性疼痛单药疗法的研发。不过，Kewalramani表示，公司将继续开展VX-993在糖尿病周围神经病变患者中的II期研究，“以进一步明确NaV1.8抑制剂在该慢性疼痛适应症中的暴露-反应关系和最大疗效”。根据临床试验数据库（ClinicalTrials.gov），该研究预计于2026年年中完成。业绩亮点难抵管线阴霾 Vertex同日公布的2025年第二季度财报显示，公司总营收29.6亿美元，同比增长12%，超出华尔街约29亿美元的预期。囊性纤维化药物Trikafta/Kaftrio（elexacaftor/tezacaftor/ivacaftor及ivacaftor）贡献25.5亿美元；Journavx上市后的首个完整季度录得1200万美元销售额。公司重申2025年全年营收指引118.5–120亿美元不变，但未能缓解投资者对镇痛管线的担忧。药事纵横投稿须知：稿费已上调，欢迎投稿

财报突破性疗法临床3期临床2期

2025-08-04

·Firstwordpharma

Vertex's pain portfolio is pummeled with setbacks

As Vertex Pharmaceuticals works to get its pain pipeline — key for its growth aspirations — off the ground, the company was hit with a one-two punch of bad news for its programmes on Monday, sending its shares down as much as 15% during after-hours trading. With an acute pain approval already in-hand for Journavx (suzetrigine), Vertex has been exploring a potential expansion for the non-opioid drug into chronic pain, while also advancing next-generation voltage-gated sodium channel inhibitors.Investors already had concerns about whether Journavx could succeed in chronic pain after the company reported in December that the NaV1.8 inhibitor showed no significant benefit over placebo in a Phase II study in patients with painful lumbosacral radiculopathy (LSR). Also known as sciatica, LSR falls into the broad peripheral neuropathic pain (PNP) bucket (see – Spotlight On: Sciatica setback magnifies doubts about Vertex’s suzetrigine in chronic pain). Vertex CEO Reshma Kewalramani confirmed during an investor call Monday that, following an end-of-Phase II meeting with the FDA, the company will not be conducting another trial of Journavx in LSR — and a general PNP approval is, for now, off the table. "While a broad PNP label remains our goal at this time, the FDA does not see a path to a broad indication," she said. "Instead, in light of the discussions and clear agreement with the FDA regarding approval requirements for diabetic peripheral neuropathy, or DPN, we will begin a second DPN Phase III study shortly in order to secure DPN as our first PNP indication for suzetrigine." Last year, Journavx won breakthrough therapy designation from the FDA for DPN, and Vertex launched a Phase III study for the indication. Now with a second late-stage DPN study planned, the biotech expects to finish enrollment for both trials by the end of 2026. "We look forward to working with the agency to secure DPN as our first PNP indication, to expand the indication over time, and to continue to discuss a potential pathway to a broad PNP label," Kewalramani added. Next-gen NaVsVertex also revealed that a next-gen, intravenously delivered NaV1.8 inhibitor, VX-993, failed a Phase II study in patients experiencing acute pain after bunionectomy surgery. The candidate did not lead to statistically significant pain relief versus placebo, and as a result, the drugmaker is halting development as a monotherapy to treat acute pain. However, the company will continue a Phase II study of VX-993 in patients with DPN "to further define the exposure response relationship and maximal efficacy of NaV1.8 inhibitors in this chronic pain indication," Kewalramani said. According to ClinicalTrials.gov, the study is expected to finish in mid-2026. She also provided a brief update on Vertex's preclinical NaV1.7 inhibitors, saying that the company is "very encouraged" by the pain candidates' progress, and that it plans to advance programmes alone or in combination with NaV1.8 inhibitors.Revenue beatVertex also shared its second-quarter financials on Monday, and while positive, the results weren't enough to offset investor disappointment with its pain portfolio. The drugmaker brought in $2.96 billion in total revenues for the quarter, up 12% year-over-year and ahead of consensus estimates of about $2.9 billion. The bulk of its revenues came from its cystic fibrosis drug elexacaftor/tezacaftor/ivacaftor and ivacaftor, which is sold as Trikafta in the US and Kaftrio in Europe. The product raked in $2.55 billion in sales.Meanwhile, the second quarter was the first full three-month period on the market for Journavx, which had $12 million in sales.Vertex reiterated its full-year guidance on Monday, predicting revenues to fall between $11.85 billion and $12 billion for 2025.

临床2期突破性疗法临床结果临床3期财报

100 项与替扎卡托相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D11041	替扎卡托	R07AX31	DB11712

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适应症	最高研发状态	国家/地区	公司	日期
囊性纤维化	临床2期	美国	Vertex Pharmaceuticals, Inc.	2012-02-01
囊性纤维化	临床2期	加拿大	Vertex Pharmaceuticals, Inc.	2012-02-01
囊性纤维化	临床2期	德国	Vertex Pharmaceuticals, Inc.	2012-02-01
囊性纤维化	临床2期	英国	Vertex Pharmaceuticals, Inc.	2012-02-01

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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


ATS2025	N/A	-	-	Elexacaftor	窪簾餘網鬱蓋獵憲顧遞(夢範願憲蓋廠蓋範夢願) = 夢餘淵選鏇鬱襯齋窪糧製艱憲遞壓艱鏇積獵築 (遞糧廠艱襯觸襯衊鹽醖 )	-	2025-05-16
ATS2025	N/A	-	-	Tezacaftor	窪簾餘網鬱蓋獵憲顧遞(夢範願憲蓋廠蓋範夢願) = 獵繭簾顧壓襯鏇醖夢膚製艱憲遞壓艱鏇積獵築 (遞糧廠艱襯觸襯衊鹽醖 )	-	2025-05-16
Pubmed \| Am J Respir Crit Care Med	临床3期	-	506	Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA)	積觸膚壓糧積艱遞齋鬱(襯餘鑰簾構積獵蓋窪憲) = Most participants had adverse events classified as mild (12.8%) or moderate (60.7%) in severity. 廠鬱範蓋齋衊鹽願憲醖 (簾範構簾鑰壓範鹽淵願 ) 更多	积极	2025-04-10
NCT02953314 (Pubmed) 人工标引	临床3期	囊性纤维化	70	tezacaftor in combination with ivacaftor	繭觸衊衊繭鏇積築繭糧(壓醖鹽遞鑰遞顧蓋廠窪) = 夢遞顧壓餘壓餘繭觸衊鹹膚繭鑰繭鏇製夢製網 (夢膚蓋鑰繭鑰築網鑰壓 )	积极	2019-09-01
NCT02070744 (CTgov)	临床2期	囊性纤维化	40	Ivacaftor+VX-661 (PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h)	製遞醖願壓獵鏇壓簾觸 = 範憲壓餘夢鏇淵夢齋鑰觸鏇憲鏇遞範膚膚鏇顧 (範範獵構廠衊鹹網膚糧, 鑰鑰鹽艱遞網願簾築選 ~ 襯糧窪醖餘壓網選簾築) 更多	-	2018-04-13
NCT02070744 (CTgov)	临床2期	囊性纤维化	40	Ivacaftor+VX-661 (PC Phase: VX-661 100 mg qd + IVA 150 mg q12h)	製遞醖願壓獵鏇壓簾觸 = 鹹繭觸製壓餘鹽衊積獵觸鏇憲鏇遞範膚膚鏇顧 (範範獵構廠衊鹹網膚糧, 觸鏇鏇範積鹹廠窪淵範 ~ 觸糧蓋衊積製醖齋範憲) 更多	-	2018-04-13