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) and VX-118 in healthy participants and in participants with cystic fibrosis (CF).

开始日期2023-12-12

申办/合作机构

Vertex Pharmaceuticals, Inc.

NCT05822102

/ Enrolling by invitationN/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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1,044

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 ; Biffi, Arianna ; Rosazza, Chiara ; 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.

188

项与替扎卡托相关的新闻（医药）

2025-05-14

·蒲公英Ouryao

Paul's Insight｜解读国际法规药事(05.05-05.11)

◆ ◆ ◆ ◆Paul's Insight2025年5月5日-5月11日◆ ◆ ◆ ◆在过去一周（5月5日至11日），全球药事监管与行业机构动作频繁：美国FDA在“美国造”战略、海外“突袭式”检查与AI审评三大领域持续发力；美国药典USP发布年度药品短缺报告，揭示供应链隐忧；欧洲EMA启动疫苗安全审查、AI与数据五年规划，并就ATMP生产指南征求意见；英国MHRA批准甲状腺眼病新药和环保吸入器、提示囊性纤维化药物心理风险；国际协调会议ICH和行业组织PDA、ISPE则在指南培训、疫苗制造速度和数字化转型等方面提供指导和思考。美国FDA监管的“三驾马车”1. “美国造”重启：行政令与供应链安全5月5日，美国总统特朗普签署《促进国内关键药品生产的监管减负》行政命令，明确要求FDA与其他联邦机构简化审批流程、提高可预测性，同时对海外原料药生产加强监管并可能公开不合规设施名单，以削弱对外依赖、保障国家安全。行政令还指示EPA加速关键设施建设审批，旨在缩短通常需要五年甚至更长的生产线落地时间，以推动罗氏、诺华等制药巨头进一步在美投资。此外，政府考虑对进口关键药品及其活性成分实施关税，以平衡贸易逆差并迫使生产回流，但具体方案尚处于内部评估阶段。图：美国总统特朗普签署《促进国内关键药品生产的监管减负》行政命令https://www.whitehouse.gov/presidential-actions/2025/05/regulatory-relief-to-promote-domestic-production-of-critical-medicines/2. 突袭检查：消弭“双重标准”5月6日，FDA宣布扩大无预警检查在海外药品及食品生产厂的应用，把此前仅在试点国家（中国、印度）进行的项目推广至全球所有出口美国的相关设施，强调“海外企业不再享有提前通知的特权”。FDA局长Marty Makary指出，此举有助于与美国本土同等标准对标，防范供应链中可能存在的质量风险。图：FDA官宣“突袭”式检查机制https://www.fda.gov/news-events/press-announcements/fda-announces-expanded-use-unannounced-inspections-foreign-manufacturing-facilities3. AI赋能：审评流程“提速降本”5月8日，FDA发布新闻稿，宣布将在6月30日前在全机构内部署生成式AI辅助科学审评系统，目标是将原需三天的文本分析与文献检索任务压缩至数分钟，被誉为“游戏规则改变者。该系统由CDER的Jinzhong Liu副主任试点，结果显示审评效率提升逾90%，极大减轻专家负担，并可将更多精力投入到复杂临床与生物等关键问题的深入评估。未来，FDA还计划将AI工具扩展至药物安全监测和临床试验数据分析，以全面构建“智能监管”体系。图：FDA宣布全面部署AI辅助审评系统https://content.govdelivery.com/accounts/USFDA/bulletins/3df85e7美国药典USP《年度药品短缺报告》核心洞见5月7日，美国药典委员会（USP）发布《年度药品短缺报告》，指出2024年总短缺案列从2023年的125例降至98例，但长期短缺（≥1年）仍占90%以上，平均持续时间从约3年增至4年以上，且无菌注射剂占短缺总量69%。报告强调，生产地域集中度、低价仿制药利润微薄、复杂制造与质量检验难度大等因素交织，导致行业缺乏应对外部冲击（自然灾害、地缘风险、政策调整）的缓冲空间。超过40种药品短缺已超三年，其中5种无菌注射剂（如硫酸阿托品、盐酸利多卡因等）短缺达十年以上；近三分之一的短缺口服固体药物单价低于1美元，44%的短缺注射剂售价低于5美元。USP副总裁Vimala Raghavendran呼吁，通过数据共享与可视化工具，促进上下游协同，探索可持续定价与采购模式，以确保质量、韧性与创新投资三者兼顾。图：美国药典委员会（USP）发布《年度药品短缺报告》https://www.usp.org/news/long-lasting-drug-shortages-drive-average-duration-higher-disrupting-patient-care美国PDA实现“100天疫苗使命”5月7日，美国注射剂协会PDA刊文阐述了流行病防备创新联盟（CEPI）“100天使命”（100 Days Mission）的背景、目标及其在化学、制造与控制（CMC）流程中推动技术创新的具体举措，旨在通过一系列前沿工艺和平台优化项目，将新发疫苗从病原体识别到紧急使用授权的时间大幅缩短，以提升全球对未来大规模疫情暴发的快速响应能力。2021年启动的“100天使命”旨在实现在今后疫情暴发时，从病原体基因序列发布起100天内完成疫苗初步授权及早期生产。PDA文章指出，COVID-19疫情期间疫苗在326天内获批，已展示了跨平台快速开发的潜能，但要真正缩短至100天，则需在制造环节进行深度革新。PDA强调，实现“100天使命”不仅需要技术突破，还需监管机构、制造商和供应链合作伙伴在预先批准流程、全球试验网络以及多区域生产能力上的协同，方能在未来疫情来袭时做到“既快又稳”。图：PDA撰文——加速疫苗供应，以应对疾病威胁的紧急用途https://www.pda.org/pda-letter-portal/home/full-article/the-need-for-manufacturing-speed欧盟EMA监管双擎驱动1. Ixchiq疫苗安全审查5月7日，欧洲药品管理局EMA安全委员会（PRAC）针对Valneva基孔肯雅减毒活疫苗Ixchiq启动审查，因全球报告17例62–89岁接种者中出现严重不良事件（含2例死亡），建议在审查期间禁止65岁及以上人群接种，同时提醒免疫功能低下者慎用。此次审查基于临床研究数据主要来自65岁以下群体，监管机构将评估该疫苗风险收益平衡，并可能调整适应症和警示条款。图：EMA 开始审查 Ixchiq（基孔肯雅减毒活疫苗）https://www.ema.europa.eu/en/news/ema-starts-review-ixchiq-live-attenuated-chikungunya-vaccine2. AI与数据五年规划同日， EMA发布《2025–2028年药品监管中的数据与AI》工作计划，描绘了人用与兽用药品监管网络中数据互操作、质量管理与高级分析的路线图，并强调符合欧洲健康数据空间（EHDS）、互联欧洲法案与AI法案的安全伦理规范，为未来监管决策提供坚实技术支撑。该计划涵盖数据治理、资产目录、元数据标准与机器学习应用等多项重点任务，旨在缩短审批周期并提高监管透明度。图：欧盟发布AI战略，打造“未来药监”体系https://www.ema.europa.eu/en/news/ema-starts-review-ixchiq-live-attenuated-chikungunya-vaccine3. ATMP生产指南修订概念文件5月8日，EMA发布《ATMP专属GMP指南第IV部分修订概念文件》，提出将2023年新版无菌生产附录1新增要求纳入ATMP指南范畴，强调需独立评估新技术与风险管理发展，面向利益相关方开放公众咨询至7月8日。该举措有助于统一基因与细胞治疗产品的高风险制造标准，并促进企业在新技术采用时保持合规。图：欧盟GMP附录1拟纳入ATMP专属指南https://ec.europa.eu/eusurvey/runner/Concept_paper_part_IV_guidelilnes_GMP_for_ATMPs英国MHRA疗法创新与环保并进1. Teprotumumab获批：首个TED靶向疗法5月7日，MHRA批准Amgen的teprotumumab（Tepezza）用于中度至重度甲状腺眼病（TED）成人患者，填补英国在该自身免疫性疾病领域的治疗空白。该药在287例患者的四项III期试验中，每3周一次输注共8次，显著改善眼球突出与复视等症状，安全性可控。图：英国首个TED靶向疗法获批https://www.gov.uk/government/news/mhra-approves-teprotumumab-as-the-first-uk-treatment-for-adults-with-moderate-to-severe-thyroid-eye-disease-ted?2. Kaftrio心理副作用风险提示5月7日，MHRA发布药物安全更新，警示囊性纤维化三联疗法Kaftrio可能引发焦虑、抑郁、睡眠障碍等心理不良反应，截至2月25日累计300份黄卡报告，涵盖9391名患者；监管机构建议医护人员及早识别并干预相关症状。媒体报道，2024年实现年销售额超过100亿美元的药品合计达12款，这是其中一款。图：Kaftrio心理副作用风险提示https://www.gov.uk/drug-safety-update/kaftriov-ivacaftor-tezacaftor-elexacaftor-risk-of-psychological-side-effects3. 批准使用“更环保”推进剂的阿斯利康吸入器5月12日，MHRA批准阿斯利康Trixeo Aerosphere低碳推进剂版，将HFA-134a替换为近零GWP的HFO-1234ze(E)，预计2025年下半年在英上市。该举措支持英国COPD患者治疗需求，同时响应制药行业碳减排承诺，推动至2045年实现90%绝对排放减少目标。图：英国批准使用“更环保”推进剂的阿斯利康吸入器https://www.gov.uk/government/news/mhra-approves-worlds-first-low-carbon-version-of-copd-inhaler-trixeo-aerosphere国际ICH发布Q5A(R2)培训材料5月6日，ICH发布Q5A(R2)《源自人或动物细胞系的生物技术产品的病毒安全性评价》培训材料0–3模块，面向行业与监管机构详细阐释2023年修订要点，并通过案例辅助实施。图：ICH发布Q5A(R2) 培训材料https://www.ich.org/news/ich-q5r2-viral-safety-evaluation-biotechnology-products-derived-cell-line-human-or-animal?ISPE三部曲监测、数字化与Pharma 4.0™ISPE本周继续撰文，对其即将举行的2025 ISPE年会主题进行了介绍。1. 数字化路线图进展数字化转型已非生物制药行业的未来愿景，而是深刻改变制造与质量控制流程的现实力量。ISPE即将在2025年生物技术大会上，汇聚终端用户、解决方案提供商与咨询机构的经验分享，从六场数据驱动、实践导向的专题演讲中揭示行业在运营、分析、质量与合规等方面的最新进展与挑战。图：IPSE探讨数字化路线图https://ispe.org/pharmaceutical-engineering/ispeak/where-are-we-digital-road-map-find-out-2025-ispe-biotechnology2. Pharma 4.0™进程Pharma 4.0™代表制药行业的数字化与数据驱动转型，旨在通过引入先进技术与稳健数据解决方案，提升生产效率、柔性和合规性，从而更快、更可靠地将创新药物推向患者。ISPE年会上将设立ATMP、数字转型、运营卓越等五大分会，搭建对话平台，推动合规与创新协同发展。图：IPSE探讨Pharma 4.0https://ispe.org/pharmaceutical-engineering/ispeak/journey-pharma-40tm-are-we-there-yet结语从简化审批到突袭检查，从AI加持到数据驱动，从安全审查到环境可持续，各方监管与行业机构正跨界协同，以应对复杂多变的全球医药挑战。展望未来，随着AI与大数据持续渗透监管决策，全球药事监管生态可能会将更加高效、透明与韧性，为保障药品质量安全及公众健康提供坚实支撑。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月16日 | 线上 | VHP生物指示剂的应用5月15日-16日 | 线下 | 2025中国制药工程设备产业大会5月20日 | 线上 | “药品注册申报中的质量研究”系列课程—“显微拉曼技术在药物制剂逆向工程中的应用”5月22日 | 线下 | 2025中国无菌药品制造创新发展论坛5月28日 | 线上 | 计算机化系统合规高效管理分享6月25日 | 线下 | 2025 CPHI 生物药商业化生产与创新论坛——分论坛：抗体药物生产能力建设研讨会6月25日 | 线下 | 2025 CPHI 生命科学与技术创新发展论坛——论坛二：细胞药物质量控制法规与技术论坛

疫苗高管变更加速审批

2025-05-05

·药明康德

5款已获FDA批准！今年有望上市的10款潜在重磅疗法最新进展一览

编者按：今年初，行业媒体Evaluate发布报告，梳理了预计于2025年上市的关键创新疗法。随着2025年进程已过三分之一，我们也对这些潜在重磅疗法的最新进展进行回顾。在报告列出的十项疗法中，有五款为小分子药物，充分体现了小分子药物在2025年生物医药创新格局中所占据的重要地位。在这五款小分子潜力疗法中，药明康德化学业务平台很高兴为其中两款提供了赋能服务，助力合作伙伴加速创新成果转化。面向未来，药明康德将持续发挥平台优势，携手全球客户，推动更多创新疗法早日惠及全球患者。图片来源：123RF在这份报告中所提到的10款潜在重磅疗法当中，已有5款率先获得美国FDA批准，包含由Vertex Pharmaceuticals所开发，在去年底获批用以治疗囊性纤维化（CF）患者的Alyftrek；由阿斯利康（AstraZeneca）与第一三共（Daiichi Sankyo）联合开发用以治疗乳腺癌的Trop2靶向抗体偶联药物（ADC）Datroway（datopotamab deruxtecan），以及同样由Vertex Pharmaceuticals所开发、用于治疗急性疼痛的Journavx（suzetrigine）则在今年1月分别接连获得FDA批准；而由GSK所开发的脑膜炎球菌疫苗Penmenvy（MenABCWY）则在2月获批；强生开发的抗体疗法Imaavy（nipocalimab）在4月底获批治疗全身性重症肌无力（gMG）。值得注意的是，在这些潜在重磅疗法当中，有不少具有显著的创新性。例如，由GSK所开发的depemokimab是在3期试验中进行评估的首个白细胞介素5（IL-5）靶向超长效生物制品。根据新闻稿，该疗法可能成为仅须每6个月给药一次、用于治疗严重哮喘的首个获批超长效生物制品。而Vertex Pharmaceuticals的新闻稿则指出，其NaV1.8抑制剂suzetrigine为二十多年来首个用于治疗急性疼痛的新机制药物。此外，强生（Johnson & Johnson）旗下新生儿Fc受体（FcRn）靶向单抗nipocalimab具有治疗多种免疫疾病的潜力，获批治疗gMG之外，它还是在干燥综合征（Sjögren’s disease，SjD）领域获得突破性疗法认定（BTD）的首款疗法。▲2025年10款潜在重磅疗法一览（图表来源：根据参考资料[1]整理制表）在列出的10款潜在重磅疗法中，小分子药物占据一半（5款，占比50%）；单克隆抗体疗法占据2款（占比20%）；而抗体偶联药物（ADC）、多肽药物及疫苗各自占据1款（各占比10%）。这一分布进一步印证了小分子药物在2025年生物医药创新版图中举足轻重的地位。2025年的十款潜在重磅疗法疗法：Alyftrek（vanzacaftor/tezacaftor/deutivacaftor）公司：Vertex Pharmaceuticals研发状态：2024年12月20日获美国FDA批准Alyftrek是一款下一代囊性纤维化跨膜电导调节因子（CFTR）调节剂三联疗法，该疗法在去年12月获FDA批准用于治疗6岁及以上、携带至少一个F508del突变或其他对Alyftrek有应答的CFTR基因突变的囊性纤维化患者。在两项随机双盲3期临床试验中，接受治疗24周后，基于一秒用力呼气量百分比（ppFEV1）数值的评估，Alyftrek的疗效与获批CF疗法Trikafta相当，达到试验的主要终点。此外，Alyftrek在降低汗液氯化物（SwCl）水平方面与活性对照药物相比表现出优效性，这是两项3期试验的关键次要终点之一。▲Alyftrek在SKYLINE 102和SKYLINE 103试验中达到主要终点（图片来源：Vertex公司官网）疗法：Datroway（datopotamab deruxtecan，Dato-DXd）公司：阿斯利康、第一三共研发状态：2025年1月17日获美国FDA批准Datroway是一款由人源化、靶向Trop2的单克隆抗体与创新DNA拓扑异构酶I抑制剂（DXd），通过可裂解的四肽连接子偶联生成的ADC。该疗法在今年1月获FDA批准上市，用于治疗无法切除或转移性HR阳性、HER2阴性的成年乳腺癌患者，这些患者曾接受过内分泌疗法和化疗。美国FDA的批准主要基于TROPION-Breast01临床3期试验的结果。分析显示，Datroway治疗组的中位无进展生存期（PFS）为6.9个月（95% CI：5.7，7.4），化疗组为4.9个月（95% CI：4.2，5.5），即Datroway相较于化疗，可将患者的疾病进展或死亡风险降低37%（HR=0.63，95% CI：0.52，0.76，p<0.0001）。Datroway与化疗组患者的确认客观缓解率（ORR）分别为36%（95% CI：31，42）和23%（95% CI：19，28）。今年1月，美国FDA授予Datroway优先审评资格用于治疗局部晚期或转移性表皮生长因子受体突变（EGFRm）的非小细胞肺癌（NSCLC）成人患者，预计将在2025年第三季度完成审评。疗法：Journavx（suzetrigine）公司：Vertex Pharmaceuticals研发状态：2025年1月30日获美国FDA批准Suzetrigine是一种口服选择性NaV1.8抑制剂，与其它NaV离子通道相比，它对NaV1.8具有高度选择性。根据关键性3期临床试验的积极结果，suzetrigine在急性疼痛治疗中表现出显著效果。具体来说，在接受腹部整形手术（abdominoplasty）患者中进行的3期临床试验结果显示，suzetrigine达到了试验的主要终点，与安慰剂相比，药物组患者48小时内疼痛强度差异的时间加权总和评分（SPID48）显著降低，SPID48的平均差异为48.4（95% CI：33.6，63.1；P<0.0001）。此外，患者在手术48小时后的数值疼痛评分量表（NPRS）评分与基线相比也显著降低。而在接受拇囊炎切除手术（bunionectomy）的患者中，该疗法亦达成主要终点，SPID48的平均差异为29.3（95% CI：14.0，44.6；P=0.0002）。▲Suzetrigine达成试验主要终点（图片来源：Vertex官网）疗法：Aficamten公司：Cytokinetics研发状态：PDUFA日期为2025年12月26日Aficamten是一种下一代心肌球蛋白抑制剂，用于治疗梗阻性肥厚型心肌病（HCM）。该药物的上市申请主要基于关键性3期临床试验SEQUOIA-HCM结果。分析显示，与安慰剂相比，aficamten治疗24周显著提高了患者的运动能力。通过心肺运动试验（CPET）测得的峰值氧气摄取量（pVO2）与基线相比，在aficamten治疗组增加了1.8 ml/kg/min，而安慰剂组为0.0 ml/kg/min。此外，接受aficamten治疗的患者在10个预先设定的次要终点中，均观察到了统计学上显著的改善。FDA曾于2021年12月授予aficamten治疗梗阻性HCM的突破性疗法认定。▲Aficamten达到SEQUOIA-HCM试验的主要终点（图片来源：Cytokinetics官网）疗法：Brensocatib公司：Insmed研发状态：PDUFA日期为2025年8月12日Brensocatib是一种口服、可逆性DPP1小分子抑制剂。DPP1是一种酶，负责激活中性粒细胞中的中性粒细胞丝氨酸蛋白酶（NSPs）。Brensocatib可通过抑制DPP1降低NSPs的活化，从而降低炎症性疾病（如支气管扩张）的组织损伤。这款疗法已经获得美国FDA授予的突破性疗法认定，用于治疗非囊性纤维化支气管扩张。一项包含1680名成人和41名青少年非囊性纤维化支气管扩张症患者的临床试验结果显示，试验达到主要终点，10 mg和25 mg剂量的brensocatib分别将患者的年化肺部症状恶化（PE）率降低21.1%和19.4%，统计学显著并具有临床意义。▲Brensocatib的3期临床试验疗效数据（图片来源：参考资料[4]）疗法：Tolebrutinib公司：赛诺菲（Sanofi）研发状态：PDUFA日期为2025年9月28日Tolebrutinib是一款在研口服、能穿越血脑屏障且具有生物活性的布鲁顿氏酪氨酸激酶（BTK）抑制剂，它能够达到足以调节B淋巴细胞和疾病相关小胶质细胞的脑脊液浓度。临床3期试验结果表明，tolebrutinib与安慰剂相比，可将非复发性继发进展型多发性硬化（nrSPMS）出现6个月确认残疾进展（CDP）的时间延缓31%（HR=0.69；95% CI，0.55-0.88；p=0.0026）。进一步的次要终点分析显示，接受tolebrutinib治疗的参与者中，确认残疾获得改善的患者比例（10%）是安慰剂组（5%）的两倍（HR=1.88；95% CI，1.10-3.21）。Tolebrutinib目前正在多个3期临床试验中评估其治疗多种类型多发性硬化的潜力。该疗法在2024年12月获得FDA授予突破性疗法认定，用于治疗nrSPMS成年患者。▲Tolebrutinib分子结构（图片来源：PubChem）疗法：玛仕度肽（Mazdutide，IBI362）公司：信达生物（Innovent）、礼来（Eli Lilly and Company）研发状态：在中国用于治疗肥胖与2型糖尿病治疗的监管申请结果预定分别在2025年上半年与下半年公布玛仕度肽是信达生物与礼来共同推进的一款胰高血糖素样肽-1受体（GLP-1R）和胰高血糖素受体（GCGR）双重激动剂。之前发布于eClinicalMedicine期刊上的一篇研究结果显示，接受每周一次9 mg玛仕度肽皮下注射治疗的受试者从基线至第12周体重的平均百分比变化为-11.7%，接受安慰剂治疗的受试者为-1.8%（估计治疗差异：-9.8%；95% CI：-14.4，-5.3；P=0.0002）。安全性方面，玛仕度肽的耐受性良好，并具有良好的安全性。未报告严重不良事件。所有治疗相关不良事件均为轻度或中度。疗法：Depemokimab公司：GSK研发状态：PDUFA日期为2025年12月16日Depemokimab是在3期试验中进行评估的首个白细胞介素5（IL-5）靶向超长效生物制品。SWIFT-1和SWIFT-2临床3期试验结果显示，试验达主要终点，depemokimab可显著降低严重哮喘患者的哮喘发作率达54%（HR=0.46，95% CI：0.36–0.59，p<0.001），这些患者具有2型炎症特征（以血液嗜酸性粒细胞计数为标志）。此外，试验亦达成次要终点。与安慰剂相比，患者需要住院或急诊救治的临床显著恶化减少达72%（HR=0.28，95% CI：0.13–0.61，p=0.002）。GSK预计在2025年启动该疗法用于治疗慢性阻塞性肺病（COPD）患者的3期临床试验。根据新闻稿，depemokimab可能成为仅须每6个月给药一次、用于治疗严重哮喘的首个获批超长效生物制品。疗法：Penmenvy（MenABCWY）公司：GSK研发状态：2025年2月14日获美国FDA批准五种脑膜炎球菌血清群（A，B，C，W和Y）占全球大部分地区的几乎所有侵袭性脑膜炎球菌病病例。GSK的五合一MenABCWY疫苗结合已获批的脑膜炎疫苗Bexsero和Menveo的抗原成分，可针对这五种血清群的脑膜炎球菌保护接种者。将两种有效疫苗合并为一种的目的是帮助简化免疫接种计划，进而增加疫苗覆盖率，帮助减少疾病的总负担。在3期试验中，MenABCWY疫苗成功达到所有主要终点，包括在免疫原性方面不劣于一剂GSK的A、C、Y和W群脑膜炎球菌疫苗，以及在对110种不同的B群脑膜炎球菌侵袭性菌株产生的免疫反应上，不劣于两剂GSK的B群脑膜炎球菌疫苗。此外，该疫苗显示出良好的耐受性，其安全性与上述两种疫苗一致。疗法：Nipocalimab公司：强生研发状态：2025年4月29日获美国FDA批准Nipocalimab是一款潜在“best-in-class”，靶向新生儿Fc受体的抗体疗法。它通过与FcRn结合，让被单核细胞和内皮细胞摄入的自身抗体不会重新释放到血液中，而是在细胞内被降解。已有研究显示，它可以将血液循环中包括致病自身抗体在内的IgG水平降低超过75%。它在4月29日获FDA批准，治疗抗AChR、抗MuSK抗体阳性的全身性重症肌无力（gMG）成人与12岁以上儿童患者。强生公司还在罕见自身抗体疾病、母体同种抗体介导的母胎疾病和风湿病领域探索nipocalimab的潜力。它在去年11月获得FDA授予突破性疗法认定，用于治疗中度至重度SjD成人患者。根据新闻稿，nipocalimab是在SjD领域获得突破性疗法认定的首个疗法。DAHLIAS临床2期试验结果显示，nipocalimab达到主要终点，从第4周开始，15 mg/kg nipocalimab组患者就开始获得缓解，并在整个24周的治疗期间持续增加。在第24周，与安慰剂相较，15 mg/kg组受试者在主要终点上，显示出平均系统性疾病活动程度相对于安慰剂超过70%的改善。▲DAHLIAS研究疗效结果摘要（图片来源：参考资料[5]）参考资料：[1] Top 10 most anticipated drug launches of 2025. Retrieved April 27, 2025 from https://www.fiercepharma.com/marketing/top-10-most-anticipated-drug-launches-2025[2] Insmed Provides Business Update at 43rd Annual J.P. Morgan Healthcare Conference. Retrieved January 21, 2025 from https://filecache.investorroom.com/mr5ir_insmed/371/2025-01-10-Insmed-Provides-Business-Update-at-43rd-Annual-J-P-Morgan-Healthcare-Conference.pdf[3] Delivering strong and sustained momentum. Retrieved January 21, 2025 from https://www.gsk.com/media/11750/gsk-2025-jpm-conf-presentation-14-january-2025.pdf[4] Insmed Announces Positive Topline Results from Landmark ASPEN Study of Brensocatib in Patients with Bronchiectasis. Retrieved May 28, 2024, from https://www.prnewswire.com/news-releases/insmed-announces-positive-topline-results-from-landmark-aspen-study-of-brensocatib-in-patients-with-bronchiectasis-302156279.html[5] LBA0010 EFFICACY AND SAFETY OF NIPOCALIMAB, AN ANTI-FcRn MONOCLONAL ANTIBODY, IN PRIMARY SJOGREN’S DISEASE: RESULTS FROM A PHASE 2, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY (DAHLIAS). Retrieved November 12, 2024 from https://ard.bmj.com/content/83/Suppl_1/240.2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

上市批准疫苗抗体药物偶联物临床3期突破性疗法

2025-04-28

·investors.vrtx.com

Vertex Receives CHMP Positive Opinion for ALYFTREK®

- ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor) recommended for people with CF ages 6 years and older with at least one non-class I mutation in the CFTR gene - - In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and superior compared to KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) at reducing sweat chloride - LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor)for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Our goal has always been to serially innovate to help people with cystic fibrosis live healthier and longer lives. If approved, this new medicine would be indicated for people with CF ages 6 years and older with at least one non-class I mutation, meaning more patients would be eligible for a medicine that gets them closer to normal levels of sweat chloride,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “CFTR modulators have already revolutionized the way we treat CF and I am encouraged that, if approved, this medicine could advance CF treatment even further,” said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin. “The results we saw from the two deutivacaftor/tezacaftor/vanzacaftor Phase 3 clinical trials were motivating as they showed non-inferiority in ppFEV1 and superior improvement of sweat chloride levels compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.” ALYFTREK® is currently licensed in the U.S. and UK and is under regulatory review in Canada, Switzerland, Australia and New Zealand. About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease. About ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic, M.D., and Professor Marcus A. Mall, M.D., in this press release, statements regarding the expected eligible patient population for ALYFTREK, if approved, and statements regarding the potential benefits of ALYFTREK. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities in the EU may not approve ALYFTREK on a timely basis or at all, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Vertex Pharmaceuticals IncorporatedInvestors:InvestorInfo@vrtx.com Media:mediainfo@vrtx.comorInternational: +44 20 3204 5275

临床结果临床3期上市批准

100 项与替扎卡托相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11041	替扎卡托	R07AX31	DB11712

研发状态

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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	窪艱齋廠衊獵憲簾構簾(壓艱製願選艱壓齋繭鬱) = 壓膚夢遞鏇鬱壓憲襯簾廠鑰蓋築膚鬱觸膚餘餘 (獵繭願製選簾製選構築 )	-	2025-05-16
ATS2025	N/A	-	-	Tezacaftor	窪艱齋廠衊獵憲簾構簾(壓艱製願選艱壓齋繭鬱) = 淵繭網憲範衊觸廠簾襯廠鑰蓋築膚鬱觸膚餘餘 (獵繭願製選簾製選構築 )	-	2025-05-16
ATS2025	N/A	F508del allele \| HbA1c \| liver function parameters ... 更多	85	Elexacaftor/Tezacaftor/Ivacaftor therapy	淵網簾構壓積廠遞願淵(鑰淵憲築夢齋鹽淵淵齋) = 廠簾觸夢憲窪齋鹹齋積願鏇願範製艱簾夢繭網 (簾艱積憲範窪餘齋餘築, 0.58 ~ 6.35) 更多	积极	2025-05-16
ATS2025	N/A	囊性纤维化	-	Elexacaftor/Tezacaftor/Ivacaftor (E/T/I)	艱獵鏇遞蓋遞獵鑰壓夢(構築獵窪鏇齋齋夢膚製) = 鏇齋襯壓蓋憲鏇糧鹽壓淵壓壓鏇鹽艱網獵壓築 (積醖鏇窪製遞願築壓餘 ) 更多	积极	2025-05-16
ATS2025	N/A	囊性纤维化 F508del mutation	-	Elexacaftor/Tezacaftor/Ivacaftor (ETI) Therapy	顧願憲選顧膚鏇繭鏇鹽(顧鏇繭築壓繭衊蓋鏇繭) = 廠製願製築糧鏇衊鏇夢願齋築廠積淵範鹽築網 (簾糧築範積鬱選繭鏇蓋 ) 更多	-	2025-05-16
ATS2024	N/A	囊性纤维化	-	Elexacaftor/tezacaftor/ivacaftor (ETI)	鹹衊築鏇齋網選繭繭鑰(淵廠窪窪衊艱鏇衊糧選) = 繭構膚鹽衊簾醖構鏇醖獵網構顧網顧獵鬱構築 (壓網願遞鏇齋鹽淵夢鏇, 5.0 ~ 98.2) 更多	-	2024-05-19
ATS2024	N/A	-	-	ETI (elexacaftor-tezacaftor-ivacaftor)	膚醖壓鹹積夢繭簾醖蓋(選觸簾淵選築繭淵壓鬱) = 淵顧製壓淵淵顧簾觸網鏇製製窪範觸願壓蓋憲 (夢簾窪觸鏇淵鹽鹽觸淵, 13) 更多	-	2024-05-19
ATS2024	N/A	肺外结核	-	Elexacaftor/tezacaftor/ivacaftor (ETI)	夢鑰製鏇憲鹽淵膚齋範(選蓋淵淵淵網窪夢網選) = 鹹鏇製網襯鑰鏇鏇夢獵積顧糧齋築蓋夢淵淵窪 (願觸衊鑰獵餘鑰壓膚淵 ) 更多	-	2024-05-19
ATS2024	N/A	移植肺并发症	-	Elexacaftor/Tezacaftor/Ivacaftor (ETI) + Tacrolimus	遞蓋窪築構遞鏇艱襯選(廠顧齋壓選繭糧範窪蓋) = One additional participant enrolled but discontinued ETI due to a rash attributed to the drug and did not complete PK studies 憲蓋窪構襯築獵鏇壓淵 (齋簾積餘鏇糧繭襯襯餘 )	-	2024-05-19
ATS2024	N/A	移植肺并发症	-	Elexacaftor/Tezacaftor/Ivacaftor (ETI)		-	2024-05-19
AASLD2023	N/A	囊性纤维化	154	Elexacaftor/tezacaftor/ivacaftor	鹹遞願膚遞餘窪獵憲醖(衊廠觸膚艱廠壓蓋憲餘) = 廠遞衊繭積顧窪衊遞簾夢齋膚願鹹觸窪網顧鏇 (蓋構衊網鏇醖膚網鹽夢 ) 更多	-	2023-11-10
ADA2023	N/A	囊性纤维化相关糖尿病	24	Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy	蓋選壓願窪構遞壓製衊(廠鹽蓋選鏇積獵範艱鹹) = 衊淵築觸繭鏇艱鹹選餘簾顧積餘範範網網齋淵 (齋築艱艱壓鏇餘膚觸網 ) 更多	-	2023-06-20