Novel therapy for the Long QT Syndrome based on the mechanism of action of the disease-causing mutations
Long QT syndrome type 2 (LQT2) accounts for
35% of all LQTS cases and is difficult to manage, as beta-blockers frequently fail to provide full protection. Most LQT2 patients (pts) have a Class 2 mutation, which implies defective "trafficking".
Lumacaftor (LUM) is a drug developed and currently indicated for the treatment of cystic fibrosis (CF) in patients homozygous for the F508del mutation in the CFTR gene. LUM corrects protein folding and trafficking defects of mutant and misfolded CFTR channels, restoring their cell surface expression. The investigators recently demonstrated that LUM can rescue in vitro the LQTS phenotype observed in human induced pluripotent stem cell- derived cardiomyocytes (hiPSC-CMs) from pts with LQT2 Class 2 mutations (PMID: 29020304) and in these same two patients Orkambi administrated for 7 days at the same dosage approved for cystic fibrosis showed to reduce their QTc (PMID: 30753398).
With the present phase II clinical trial (MAST2) the investigators will enroll 20 LQT2 patients (see inclusion and exclusion criteria) and they will test in vivo the efficacy of Orkambi in shortening their QTc. Patients will be admitted to hospital for a maximum of 7 days (minimum in-hospital stay based on evidence of QTc shortening). Orkambi will be administered at the dose approved for cystic fibrosis and during the entire period continuous ECG monitoring through both telemetry and 12-lead 24-hr Holter monitoring will be performed and QTc length and morphology will be analyzed.

开始日期2021-06-15

申办/合作机构

Istituto Auxologico Italiano

NCT04235140

/ Completed临床3期

A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

开始日期2020-02-24

申办/合作机构

Vertex Pharmaceuticals, Inc.

NCT03251911

/ Withdrawn临床4期IIT

An Open Label Study to Investigate the Role of Ivacaftor (VX-770) for the Treatment of Chronic Bronchitis With CFTR Dysfunction

This research study will test how well a new drug affects bronchiectasis or chronic bronchitis. The new drug, Ivacaftor (KALYDECO), is a drug that has recently been approved by the U.S. Food and Drug Administration (FDA) for patients with a lung disease called Cystic Fibrosis (CF). It has not been approved for use in patients with bronchiectasis or chronic bronchitis.

开始日期2020-01-01

申办/合作机构

The University of Alabama at Birmingham

100 项与依伐卡托/芦马卡托相关的临床结果

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100 项与依伐卡托/芦马卡托相关的专利（医药）

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102

项与依伐卡托/芦马卡托相关的文献（医药）

2025-11-01·Journal of Cystic Fibrosis

Treatment decision-making for using CFTR modulator therapy in patients with cystic fibrosis

Article

作者: Griffin, Michael ; Higgins, Mark ; Carneiro, Carlos ; Salvatore, Donatello ; Carr, Alison ; Plant, Barry J ; Wöhling, Heike ; Elborn, J Stuart ; Hammond, Fiona

BACKGROUND:

Five cystic fibrosis transmembrane conductance regulator (CFTR) modulator (CFTRm) therapies are approved for patients with cystic fibrosis (CF). Despite the established efficacy and safety of CFTRm therapy, variability exists in prescribing amongst CF physicians. Here, we describe prescriber factors influencing CFTRm use.

METHODS:

This observational study used clinical vignettes of patients with CF. Participants were adult pulmonologists and pediatricians specializing in respiratory medicine from Germany, Italy, UK, Ireland, and the Netherlands. Participants took part in a 3-step data collection process: (i) 60-minute individual telephone interview; (ii) decision exercise where physicians made a treatment decision about CFTRm on 10 clinical vignettes; and (iii) 30-minute web-assisted interview to provide rationale for treatment decisions. Univariate analysis, dimensionality reduction via factor analysis, and a random-effects logistic regression model were used to identify factors impacting CFTRm prescribing.

RESULTS:

Thirty-eight pulmonologists and 42 pediatricians provided 800 clinical decisions. Only 45 % of pulmonologists and 29 % of pediatricians prescribed a CFTRm for all eligible adult or pediatric vignettes. Pancreatic sufficiency decreased odds of prescribing a CFTRm (OR 0.106; 95 % CI 0.046, 0.244) whereas odds increased for patients' age >12 years (OR 3.779; 95 % CI 1.579, 9.042). Physician characteristics associated with CFTRm prescribing were being proactive/pushing boundaries (OR 1.772; 95 % CI 1.153, 2.722), having previously prescribed LUM/IVA (OR 2.732; 95 % CI 1.070, 6.974), and belief CFTRm therapies could improve adherence (OR 1.440; 95 % CI 0.946, 2.192).

CONCLUSIONS:

Behavioral characteristics, attitudes toward CFTRm therapies, and experience with CFTRm therapies impact physician CFTRm prescribing more than individual disease factors.

2025-09-01·DRUGS IN R&D

Demonstrating Bioequivalence for a Lumacaftor Monosubstance Formulation Versus Orkambi® (Lumacaftor/Ivacaftor) in Healthy Subjects

Article

作者: Lidington, Darcy ; Papaelias, Alexandra ; Bolz, Steffen-Sebastian

BACKGROUND AND OBJECTIVE:

Lumacaftor is an active ingredient in the US Food and Drug Administration-approved combination medication Orkambi^®, which is used for treating cystic fibrosis. Experimental evidence suggests that lumacaftor can be used as a monotherapy to improve brain perfusion and memory in heart failure. To clinically assess this therapeutic intervention, a formulation with demonstrated bioequivalence to the currently approved combination product is required.

METHODS:

This comparative bioavailability and food-effect study compared lumacaftor pharmacokinetics in healthy patients following: (i) oral administration of lumacaftor (400 mg; Test Product) or Orkambi^® (lumacaftor 400 mg/ivacaftor 250 mg; Reference Product) in the fed state and (ii) oral administration of lumacaftor (400 mg; Test Product) in the fasted to fed state. Plasma lumacaftor concentrations were measured with a standard liquid chromatography with tandem mass spectrometry approach.

RESULTS:

The "Test-to-Reference ratio" of the geometric least-square means for maximum plasma concentration and area under the curve met the Food and Drug Administration-defined criteria for bioequivalence; median times to maximum plasma concentration values were not statistically different. The "Fed to Fasted ratio" of the geometric least-square means for maximum plasma concentration and area under the curve indicated a clear food effect on bioavailability. Lumacaftor exposure was approximately two times higher when administered with fatty foods than when taken in a fasting state. The monosubstance formulation was well tolerated.

CONCLUSIONS:

We conclude that the lumacaftor monosubstance formulation delivers lumacaftor exposure that is not meaningfully different than the currently approved combination product.

CLINICAL TRIAL REGISTRATION:

ClinicalTrials.gov identifier: NCT05968612.

2025-06-01·AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Lumacaftor inhibits channel activity of rescued F508del cystic fibrosis transmembrane conductance regulator

Article

作者: Rowe, Steven M. ; Thompson, Jake ; Hagedorn, Zachary J. ; Rodriguez-Cruz, Kevin ; Wen, Hui ; Bono, Taylor R. ; McNicholas, Carmel M. ; Phillips, Aubrey J. ; Freeman, Alana J. ; Ali, Judge ; Ambrosetti, Adam D. ; Palacio, Hayes ; Wang, X. Robert ; Fu, Lianwu ; Gilliland, Stephanie D. ; Nguyen, Rodney

Small-molecule correctors bind to F508del cystic fibrosis transmembrane conductance regulator (CFTR) and restore its trafficking to the plasma membrane to function as an anion channel. Despite its high efficacy as a corrector, lumacaftor inhibits the channel opening of rescued F508del CFTR, making it a weak CFTR modulator. The current work highlights the impact of CFTR correctors on the channel activity of rescued F508del CFTR as an important variable in the efficacy of modulator therapy.

139

项与依伐卡托/芦马卡托相关的新闻（医药）

2026-03-11

·规培小生聊科研

Nature子刊 | 从培养皿到临床：类器官技术如何重塑药物研发版图

类器官技术自2009年诞生以来，已从概念验证走向临床转化，成为连接基础研究与药物开发的关键桥梁。与传统二维细胞系和动物模型相比，类器官能够更真实地重现人体组织的细胞异质性、空间结构和生理功能，为疾病机制解析、药物筛选及安全性评价提供了前所未有的研究平台。 2026年发表在Nature Reviews Drug Discovery上的这篇综述系统阐述了类器官技术的最新进展、应用现状及面临挑战，为这一领域的未来发展指明了方向。引言1. 类器官的定义与分类基本定义：类器官是由干细胞衍生的三维结构，能够重现相应组织的关键架构和功能特征主要来源：多能干细胞（PS细胞）来源：包括胚胎干细胞（ES细胞）和诱导多能干细胞（iPS细胞），通过逐步谱系定向分化形成组织干细胞（TSC）来源：直接从活检样本或原代组织建立，无需体外谱系定向2. 类器官技术的发展历程早期基础：Mina Bissell团队开创性地使用Matrigel进行上皮细胞三维培养，证明乳腺上皮细胞可形成三维腺体结构并分泌乳汁关键突破： 2006年Yamanaka发明iPS细胞技术，解决了ES细胞的伦理和来源限制 2009年首次建立肠道TSC来源类器官，展示单个Lgr5+干细胞可自我组织形成完整隐窝-绒毛结构 Yoshiki Sasai团队创建首个PS细胞来源的视网膜和中枢神经系统类器官3. 类器官与传统模型的比较优势比较维度类器官传统2D细胞系动物模型生理相关性高：模拟人体组织复杂性低：结构模拟有限高：整体生物背景，但存在种属差异通量中/高高低成本/时间中等低高可扩展性中/高高低监管接受度新兴且日益认可已确立已确立4. 核心应用价值疾病建模：遗传性疾病、癌症、感染性疾病药物筛选：高通量筛选、个性化医疗安全性评价：毒性预测、ADME研究监管趋势：FDA Modernization Act 2.0/3.0推动减少动物实验，类器官作为新替代方法（NAMs）地位提升类器官在疾病建模中的应用1. 遗传性疾病建模1.1 囊性纤维化（Cystic Fibrosis, CF）疾病机制：CFTR基因功能缺失突变导致氯离子/碳酸氢盐通道功能障碍类器官模型：肠道和气道类器官关键发现：福斯可林（forskolin）诱导的类器官肿胀实验可定量评估CFTR功能药物反应与患者临床结局（如ppFEV1、痰细胞计数）高度相关实现个性化医疗：Orkambi等CFTR调节剂的疗效预测1.2 原发性纤毛运动障碍（PCD）模型建立：患者鼻气道类器官应用价值：直接观察纤毛搏动、电子显微镜结构分析、基因治疗概念验证1.3 肾脏遗传疾病常染色体显性多囊肾病（ADPKD）：可重现囊肿形成和疾病基因特征表达常染色体隐性多囊肾病（ARPKD）：器官芯片平台模拟流体流动和囊肿发生1.4 肝脏遗传疾病 α1-抗胰蛋白酶缺乏症（AATD）：重现A1AT聚集、蛋白分泌减少和弹性蛋白酶抑制受损 Wilson病：胆管细胞类器官显示铜处理敏感性增加 Alagille综合征：延迟分化为导管细胞、管腔凋亡增加1.5 中枢神经系统遗传疾病帕金森病（PD）：中脑类器官重现多巴胺神经元发育障碍、α-突触核蛋白聚集、线粒体和溶酶体功能障碍阿尔茨海默病（AD）：APOE4变异类器官显示Aβ和磷酸化tau增加、突触完整性降低额颞叶痴呆（FTD）：MAPT突变类器官揭示tau病理进展和谷氨酸兴奋毒性机制2. 代谢性疾病建模2.1 代谢功能障碍相关脂肪性肝炎（MASH）模型特点：患者来源或代谢物诱导的肝类器官重现脂质积累、促炎通路激活和肿瘤相关标志物上调药物筛选应用：二甲双胍、L-肉碱、FGF19等疗效评估2.2 其他代谢疾病 Wolman病：FGF19通过激活FXR减少脂质储存诱导的ROS活性家族性高胆固醇血症：心脏糖苷类降低载脂蛋白B产生线粒体DNA耗竭综合征：NAD+治疗改善线粒体功能3. 慢性炎症性疾病建模3.1 炎症性肠病（IBD）关键发现： IL-22刺激结肠类器官揭示CXCR2+中性粒细胞招募机制患者来源类器官保留炎症组织转录特征自体外周血T细胞共培养模型揭示CD103和NKG2D依赖的上皮细胞损伤机制3.2 乳糜泻（CeD）模型创新：气-液界面系统保留肠道上皮和驻留免疫细胞机制发现：IL-7作为麸质诱导的致病调节因子3.3 慢性阻塞性肺疾病（COPD）表型重现：杯状细胞增生、纤毛搏动频率降低吸烟相关研究：香烟烟雾提取物诱导的类器官形成抑制4. 感染性疾病建模4.1 肠道病毒感染隐孢子虫（Cryptosporidium parvum）：首次在体外支持完整无性-有性生命周期人诺如病毒（HuNoV）：首个体外人源平台，证实组织血型抗原结合特异性轮状病毒、柯萨奇B病毒、肠道病毒：完整感染周期和抗病毒药物筛选4.2 寨卡病毒（ZIKV）关键发现：感染神经前体细胞导致小头畸形表型 NS2A蛋白破坏放射状胶质细胞增殖 NS4A/NS4B抑制AKT-mTOR信号阻断神经发生药物筛选鉴定hippeastrine hydrobromide、emricasan和niclosamide等候选药物4.3 呼吸道病毒感染 SARS-CoV-2：ACE2+纤毛细胞为主要靶点，TMPRSS2为关键激活因子流感病毒：区分人感染性和禽/猪亚型的复制能力呼吸道合胞病毒（RSV）：NS2蛋白驱动的细胞运动性和中性粒细胞招募5. 癌症建模5.1 患者来源肿瘤类器官（PDTOs）的优势可建立性：几乎所有癌种均可建立保真性：重现亲本肿瘤的组织学、遗传学和转录组特征可扩展性：可异种移植进行体内表征基因工程模型：CRISPR序列编辑致癌突变创建确定性癌症模型5.2 药物开发与个性化医疗 MCLA-158（petosemtamab）案例：完全基于结肠癌类器官生物库开发的双特异性抗体，从发现到I期临床仅30个月临床相关性：PDTO药物敏感性与患者治疗反应显著相关（胰腺癌、胃肠道癌、乳腺癌）生物库建设：多种肿瘤类型的PDTO生物库建立（胃癌、结直肠癌、胰腺癌、肝癌、乳腺癌、卵巢癌等）类器官在药物安全性评价中的应用1. 肝脏毒性模型优势：TSC和PS细胞来源肝类器官表达I/II期药物代谢酶和转运蛋白应用案例：曲格列酮、对乙酰氨基酚、曲伐沙星等肝毒性预测基因型特异性敏感性（如bosentan诱导的胆汁淤积性肝损伤）药物性磷脂质病模型优于传统HepG2细胞 AAV基因治疗载体性能评估2. 肾脏毒性模型特点：包含肾单位、集合管、肾间质和内皮细胞，自组织形成肾单位样结构毒性评估：顺铂、庆大霉素、阿霉素等诱导的剂量依赖性细胞凋亡和KIM1表达先进系统：免疫浸润、血管化肾脏类器官芯片评估T细胞双特异性抗体介导的选择性杀伤3. 胃肠道毒性腹泻预测：回肠类器官模型对31种参考药物预测准确率达90% 机制研究：阿霉素、吉非替尼诱导毒性的转录组分析免疫相关毒性：T细胞双特异性抗体的"on-target, off-tumour"效应评估4. 中枢神经系统毒性长春新碱：揭示对脑发育的神经毒性效应，涉及ECM介导信号和基质金属蛋白酶反苯环丙胺：剂量依赖性脑损伤，通过BHC110/LSD1靶向基因转录抑制紫杉醇：化疗诱导神经毒性评估5. 心脏毒性阿霉素模型：减少活力、增加LDH和BNP水平、诱导凋亡、纤维化和线粒体功能障碍 FDA召回药物筛选：类器官比2D单层更有效地检测剂量依赖性ATP耗竭和搏动中断多器官平台：肝脏-心脏共培养揭示肝脏代谢介导的心脏毒性（如氯米帕明）类器官在ADME研究中的应用1. 肝脏代谢酶表达：CYP3A4、CYP2D6等I期酶，UGT和磺基转移酶等II期酶转运蛋白：OATP1B3、ABCB1/MDR1、MRP3 应用：咪达唑仑代谢和生物利用度、香豆素和二甲双胍排泄研究2. 肠道吸收与代谢 CYP酶诱导：地塞米松、β-萘黄酮、TCPOBOP诱导药物转运：头孢羟氨苄、β-内酰胺类抗生素、环六肽的摄取研究双向转运：Transwell系统支持顶端/基底双向转运研究3. 肾脏转运与清除转运蛋白表达：OCT2、OAT1、OAT3上调功能验证：荧光罗丹明123检测OCT1/2介导的转运4. 当前局限与未来方向成熟度限制：肝和肾类器官分化成熟度不足技术挑战：需要代表体内情况的酶活性、屏障功能和足够组织量发展潜力：流体连接的多器官系统和血管化类器官解决药代动力学和组织分布挑战当前挑战与解决方案1. 技术挑战1.1 复杂性不足缺失组分：血管、免疫细胞、神经支配、细胞外基质、基质细胞系统性互作缺失：激素信号、多器官串扰、生物力学力（剪切应力、拉伸、压力）1.2 变异性和 reproducibility 来源变异：不同患者供体的遗传多样性方案差异：实验室间培养条件不一致分化异质性：批次内和批次间功能差异2. 解决方案：生物工程与自动化2.1 微流控和器官芯片技术精准控制：器官oid形状、大小和细胞数量的精确控制多腔室系统：整合血管、免疫和基质细胞实时监测：内置生物传感器检测ATP/ADP比率2.2 高通量筛选平台微孔阵列：同时生成数千个均一器官oid 微柱芯片：384孔板格式神经毒性筛选自动化液体处理：机器人介导的培养基更换、药物添加和化合物筛选2.3 成像与数据分析高内涵成像：CellInsight CX7等自动化平台实时监测生长、形态变化和药物反应人工智能辅助：计算机视觉和图像分析算法量化细胞活力、结构完整性、基因表达组织微阵列：支持AI辅助定量图像分析和分子病理学方法3. 监管挑战与机遇3.1 当前监管框架 FDA立场：不强制规定支持首次人体临床试验的研究类型（体内、体外或计算机模拟）新替代方法（NAMs）：FDA Modernization Act 2.0/3.0明确动物实验非必需 FDA替代方法计划：扩大替代方法监管使用资格认定流程3.2 验证与资格认定 OECD指导文件：GIVIMP、GD211、GD34支持器官oid的详细描述、特征化和验证 EMA安全港方法：允许生成、汇编和审查数据以定义NAMs的使用背景跨行业合作：生物工程师、生物学家、毒理学家和病理学家协作开发3.3 未来展望渐进式整合：将器官oid作为NAMs纳入监管申报，替代、减少和优化动物模型平衡创新与安全：拥抱创新方法同时维持药物评估的严格标准结论与展望1. 变革性潜力类器官技术代表了向更准确、更患者相关的临床前研究模型的变革性飞跃。相比传统2D细胞培养和动物模型，类器官提供了更生理相关的人体组织复杂性重现平台。2. 持续挑战可扩展性和可重复性：特别是高通量筛选背景下的进一步优化需求预测能力提升：癌症、神经退行性疾病、慢性炎症和代谢性疾病的复杂性技术整合：血管化、免疫系统整合、多器官建模与单细胞组学、高速成像和AI支持数据分析的结合3. 未来方向标准化自动化平台：持续开发和验证监管框架完善：促进行业采用临床转化验证：前瞻性随机临床试验评估预测价值4. 最终愿景随着技术成熟，基于类器官的平台有望成为药物发现流程的组成部分，弥合基础科学与临床结果之间的差距，最终革命性地改变我们理解和治疗疾病的方式。

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2026-02-13

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福泰制药2025年财报：TRIKAFTA/KAFTRIO营收103亿美元

2026年2月12日，福泰制药公布了其2025年第四季度及全年财务业绩。报告清晰地展示了一家行业领先的生物技术公司在核心业务稳健增长的同时，正成功地向多疾病领域治疗平台转型。全年总收入达120.013亿美元，较2024年的110.201亿美元增长9%；第四季度总收入为31.900亿美元，同比增长10%。更为显著的是，公司盈利能力实现根本性扭转，全年GAAP净利润从2024年的净亏损5.356亿美元转为盈利39.532亿美元。这份成绩单不仅体现了其王牌囊性纤维化（CF）业务的持续韧性，更凸显了新上市产品CASGEVY和JOURNAVX带来的早期贡献，以及一个广阔的中后期研发管线所蕴含的巨大潜力。一、财务表现：收入稳健增长，盈利能力根本性改善1. 收入增长动力：CF基石稳固，新业务贡献初显福泰制药2025年120.013亿美元的总收入增长，主要驱动力来自CF疗法的持续表现以及三款新上市产品的早期贡献。其中，产品净销售额为119.706亿美元。 CF产品线：核心产品TRIKAFTA/KAFTRIO全年收入103.127亿美元，依然是公司的收入压舱石。新一代CFTR调节剂ALYFTREK自上市后快速放量，全年贡献收入8.378亿美元，成为重要的增长增量。其他CF产品（KALYDECO, ORKAMBI, SYMDEKO/SYMKEVI）收入8.201亿美元。新上市产品：基因编辑疗法CASGEVY（治疗SCD和TDT）全年收入1.158亿美元；口服非阿片类止痛药JOURNAVX（suzetrigine）全年收入5960万美元。这两款产品的收入虽占总收入比例尚小，但标志着公司成功迈出了业务多元化的关键第一步。分区域看，美国市场表现尤为强劲，全年收入增长13%至75.5亿美元，主要得益于强劲的CF患者需求、ALYFTREK的上市、CF产品实际净价格的提高以及CASGEVY和JOURNAVX的贡献。美国以外市场收入增长3%至44.5亿美元。2. 利润与现金流：从巨额收购影响中恢复，盈利强劲 2025年最突出的财务变化是利润表的根本性改善。全年GAAP净利润39.532亿美元（每股收益15.32美元），而2024年为净亏损5.356亿美元（每股亏损2.08美元）。这一巨大逆转的核心原因是2024年第二季度因收购Alpine Immune Sciences产生了高达46.284亿美元的“收购的在研研发资产（Acquired IPR&D）”费用，而2025年该项费用已恢复正常水平，仅为1.33亿美元。从更能反映核心经营状况的非GAAP指标看，公司2025年非GAAP净利润为47.470亿美元（每股18.40美元），较2024年的1.106亿美元实现了数量级的飞跃。这得益于收入增长以及运营费用的有效管理。全年非GAAP研发、收购IPR&D及销售管理费用合计51.182亿美元，远低于2024年（含Alpine收购费用）的88.217亿美元。公司的财务状况非常健康。截至2025年12月31日，现金、现金等价物及有价证券总额为123亿美元，较2024年底的112亿美元有所增加，为公司持续进行研发投资和业务拓展提供了充足弹药。二、核心业务板块：从CF领导者到多疾病领域开拓者1. 囊性纤维化（CF）：持续创新，巩固绝对领导地位福泰在CF领域超过20年的深耕构筑了极高的护城河。其CFTR调节剂组合在核心市场可覆盖近95%的CF患者。2025年的重点进展包括： ALYFTREK市场渗透：已在美、英、欧盟等多国获批并获报销。针对2-5岁儿童的关键研究显示，治疗24周后平均汗液氯离子降低9.6 mmol/L，65%的儿童达到正常值（<30mmol/L）。公司预计在2026年上半年向全球监管机构提交该年龄组的批准申请。患者群体扩张：公司预计，通过新药批准、报销协议达成、治疗更低龄患者、患者生存期延长以及市场地域扩张，使用其CF药物的患者数量将持续增长。2. 基因编辑疗法CASGEVY：商业化稳步推进，未来可期作为全球首个获批的CRISPR/Cas9基因编辑疗法，CASGEVY在2025年实现了重要的商业化起步。收入与患者数据：全年收入1.158亿美元，第四季度收入5430万美元。这反映了2025年全年共有64名患者接受了CASGEVY输注，其中第四季度有30名。全球范围内，2025年有147名SCD或TDT患者为接受CASGEVY治疗完成了首次细胞采集。市场准入与拓展：截至2025年底，美国约90%的患者已获得CASGEVY的报销覆盖，该药在英国、意大利、沙特阿拉伯等多个国家也已获报销。针对5-11岁儿童的研究数据积极，公司计划在2026年上半年提交该年龄组的全球监管申请。3. 疼痛管理产品JOURNAVX：上市首年快速放量 JOURNAVX作为首款口服、选择性、非阿片类Nav1.8疼痛信号抑制剂，上市后取得了强劲开端。市场接受度：自2025年3月初在药房上市至年底，在医院和零售渠道已开具并调配了超过55万张处方。保险覆盖：截至2026年1月，公司已确保与全美三大药房福利管理公司（PBM）的全部合作，覆盖超过2亿商业和政府保险参保人，占美国受保人口的三分之二。此外，21个州的医疗补助计划已提供覆盖。三、研发管线：丰富的“催化剂”储备，定义未来增长曲线福泰制药的核心竞争力在于其深度和广度俱佳的研发管线。2025年，公司在中后期临床项目上取得多项关键进展。1. 临近商业化的重磅候选药 Povetacicept（IgA肾病等）：这款BAFF/APRIL双重抑制剂具有“管线中产品”的潜力。2025年第四季度，公司已启动向美国FDA滚动提交生物制品许可申请（BLA），用于加速批准治疗IgA肾病（IgAN），并计划在2026年上半年完成提交。FDA已授予其针对IgAN的突破性疗法认定。2. 中后期关键项目推进 APOL1介导的肾脏病（AMKD）：口服小分子抑制剂inaxaplin的II/III期AMPLITUDE试验已完成中期分析队列的入组，预计在2026年底或2027年初分享中期分析数据。 1型糖尿病（T1D）：干细胞衍生胰岛细胞疗法zimislecel的I/II/III期研究已完成患者入组。该疗法已获得FDA的再生医学先进疗法（RMAT）和快速通道认定。其他领域：公司在周围神经性疼痛（PNP）、原发性膜性肾病（pMN）、常染色体显性多囊肾病（ADPKD）、1型强直性肌营养不良（DM1）等多个疾病领域均有项目进入概念验证或关键研究阶段。四、未来展望：指引乐观，增长路径清晰基于当前业务势头，福泰制药对2026年给出了积极且具体的财务指引：总收入：预计在129.5亿美元至131亿美元之间。非CF产品收入：预计将达到5亿美元或更多，这主要来自CASGEVY在全球ATC网络输注患者的增加，以及JOURNAVX上市第二年的处方和收入增长。费用投入：公司计划继续加大研发和商业化投入，预计全年非GAAP研发、收购IPR&D及销售管理费用在56.5亿至57.5亿美元之间，以支持众多中后期临床开发项目。总结纵观福泰制药2025年全年表现，可以得出以下结论：公司正处于一个“稳健基本盘”与“广阔新边疆”协同发力的理想发展阶段。其CF业务凭借ALYFTREK等产品的迭代和患者群体的扩张，继续提供强劲且可预测的现金流。与此同时，CASGEVY和JOURNAVX的成功上市及早期商业化表现，有力证明了公司将科学创新转化为商业价值的能力，多元化转型已初见成效。更为重要的是，公司在IgAN、AMKD、T1D等重大疾病领域布局了数量可观、阶段成熟的研发管线，其中多个项目有望在近期成为新的增长引擎。首席执行官Reshma Kewalramani博士将2025年定义为“强劲收入增长、商业多元化和管线推进的一年”，这一判断在财报数据中得到了充分印证。凭借深厚的财务实力、已被验证的商业化能力以及行业领先的研发创新能力，福泰制药正朝着其“为严重疾病患者创造变革性药物”的长期目标稳步迈进，并为股东创造可持续的长期价值。相关阅读：默沙东公布2025年业绩，总营收增长1%，K药大卖317亿美元艾伯韦2025年业绩：营收增长9%至612亿美元，利生奇珠单抗大卖176亿美元快速觉醒的巨人：阿斯利康2025年总营收增长8%达587亿美元诺和诺德2025年财报：司美格鲁肽虽大卖345亿美元，但依旧无缘药王辉瑞2025年成绩单：营收同比下降2% 诺华2025年销售额增长8%达545.32亿美元，诺欣妥77.5亿美元安进2025年营收增长10%达368亿美元，礼来2025年成绩单：营收大涨45%至652亿美元，替尔泊肽大卖365亿美元赛诺菲公布2025年业绩，全年营收增长9.9%至436亿欧元，Dupixent大卖157亿欧元罗氏公布2025年的业绩，销售额增长7%达615亿瑞郎 GSK：总营收327亿英镑，同比增长4% 吉利德2025年业绩：营收294亿美元，净利润85亿美元渤健业绩：总营收99亿美元，净利润12.9亿美元 BMS公布2025年业绩：阿哌沙班依然坚挺，销售额达144亿美元再生元2025年业绩：总营收143亿美元，净利润45亿美元强生2025年业绩稳健增长，总营收942亿美元，药品营收604亿美元立即扫码加入药事纵横交流群

财报基因疗法并购

2026-02-12

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采用MonolixSuite软件发表的部分参考文献汇总（2024年7月-2025年12月）

采用MonolixSuite软件发表的部分参考文献汇总（2024年7月-2025年12月）导读凡默谷技术部挑选了2024年7月—2025年12月全球采用MonolixSuite软件发表或涉及MonolixSuite综述的部分应用文章。希望对您的业务或专业学习有所帮助。本公众号不提供文章的原文下载，如您想了解某篇文章的详情，请下载《采用MonolixSuite软件发表的部分参考文献汇总列表 2024年7月-2025年12月》PDF版文件，有对应文章的介绍链接。2024年7月-2025年12月 PDF版文献汇总列表下载(复制链接到浏览器）https://www.jianguoyun.com/p/DfVhFxUQ_a64Bxih9ZkGIAA访问密码：HdX0tA01中国用户发表的部分文章或涉及到的综述用于预测拓扑异构酶抑制剂ADC药物在小鼠及人体内的药代动力学、肿瘤生长抑制效应及毒性风险的 PBPK-PD 模型PBPK-PD model for predicting pharmacokinetics, tumor growth inhibition, and toxicity risks of topoisomerase inhibitor ADCs in mice and humans西维来司他钠在中国重症肺炎患者中的群体药代动力学研究Population pharmacokinetics of sivelestat in Chinese patients with severe pneumoniaZhang X., Hu H., Li Z., Zhang P., Pan L., Wang L., Mao J., Li F., Zhang L. Fundamental and Clinical Pharmacology. 2025 39:2 Article Number e70001亚胺培南在感染性实体瘤患者中的群体药代动力学：一项真实世界研究Population pharmacokinetics of imipenem in solid tumor patients with infections: A real-world studyXu T., Zou T., Zhang J., Li Z., Li F., Du J., Hao Z. Journal of Global Antimicrobial Resistance. 2025 45 (107-114)PopPK和PBPK模型指导美罗培南在重症患儿增强肾脏清除率下的剂量优化PopPK and PBPK Models Guide Meropenem Dosing in Critically Ill Children with Augmented Renal ClearanceLiu Y., He H., Zhang S.-S., Zhou J., Zhu J.-W., Xu J., Miao H.-J., Chen J.-H., Hao K. Pharmaceutics. 2025 17:12 Article Number 1544达泊西汀在健康中国男性受试者中的群体药代动力学Population Pharmacokinetics of Dapoxetine in Healthy Chinese Male SubjectsPu L., Wu T., Bai F., Lu H., Wang Y., Gao Y., Qiu W. Journal of Clinical Pharmacology. 2025丙泊酚注射液的群体药代动力学研究进展陈浩，陈建恒，吕洁，严泽林，卞民亮，陆程灿.《临床药物治疗杂志》. 第24 卷第1 期, 2026年1月02其他国家用户发表的部分文章或相关的综述基于模型的荟萃分析应用 MonolixSuite：针对纵向分类与连续数据的教程Model-Based Meta-Analysis With MonolixSuite: A Tutorial for Longitudinal Categorical and Continuous DataBracis C., Taneja A., Lyauk Y.K., Barcomb H., de la Peña A., Cellière G.CPT: Pharmacometrics and Systems Pharmacology2026 15:2 Article Number e70158 高强度间歇性运动对二甲双胍在健康男性的药代动力学影响——基于PopPK模型的评估Effect of high-intensity interval exercise on metformin pharmacokinetics in healthy men, assessed through a population pharmacokinetic modelNikolaidis S., Kosmidis I., Papadopoulos S., Lioupi A., Gandanidou M., Gika H., Dokoumetzidis A., Theodoridis G., Mougios V. British Journal of Pharmacology. 2026 183:3 (438-452) 抗胸腺细胞球蛋白在髓消性造血干细胞移植中的PopPK与最佳药物暴露Population pharmacokinetics and optimal exposure of anti-thymocyte globulin in myeloablative hematopoietic cell transplantationGhazal H., Leuchter S., Ngo R., Kinzel M., Dabas R., DharmaniKhan P., Khan F.M., Jamani K., Li N., Kiang T.K., Storek J. Transplantation and cellular therapy. 2026 他克莫司口服混悬液在造血干细胞移植患儿中的PopPK及生物利用度研究Population pharmacokinetics and bioavailability of tacrolimus oral suspension in children with haematopoietic stem cell transplantBrstilo L., Ibarra M., Juliá A., Testard J., Salgueiro R., Mendoza A., Pérez C., Licciardone N., Marquet P., Staciuk R., Buontempo F., Schaiquevich P. British Journal of Clinical Pharmacology. 2026 92:1 (210-219) 从 AUC/MIC 到 AUCss 与 Cmin：通过模型引导米卡芬净在危重患者治疗中的精准给药的优化From AUC/MIC to AUCss and Cmin: Optimizing Micafungin Therapy in the Critically Ill through Model-Informed Precision DosingBerrah R., Saint Marcoux F., Monchaud C., Cointault O., Conseil M., Jaber S., Jung B., Woillard J.B. AAPS Journal2026 28:1 Article Number 18阿莫西林在人乳中的PopPK建模——ConcePTION 项目的一项贡献Population pharmacokinetic modelling of amoxicillin in human breast milk—A contribution from the ConcePTION projectBaklouti S., Rigourd V., Panchaud A., Nordeng H., Allegaert K., Annaert P., Huang M.-C., Monfort A., Guidi M., Gandia P. British Journal of Clinical Pharmacology. 2026重症监护病房（ICU）收治的医院获得性肺炎患者中，他唑巴坦联合哌拉西林给药的关键阈值目标达标率Critical threshold target attainment rates for tazobactam combined with piperacillin among patients admitted to the ICU with hospital-acquired pneumonia含删失协变量和测量误差的广义线性混合模型及其在 HIV/AIDS 研究中的应用Generalized Linear Mixed Models with Censored Covariates and Measurement Errors, with Applications in HIV/AIDS StudiesGao S., Wu L. Statistics in Biosciences. 2025 17:3 (832-854)神经重症监护人群中左乙拉西坦给药方案的优化：Neuro-ARC 研究Levetiracetam Dosing Optimization in Neurocritical Care Population: Neuro-ARC StudyKharouba M., Cook A.M., Bastin M.L.T., Kutsogiannis D.J., Mahmoud S.H. Neurocritical Care. 2025 43:3 (887-901)结合人胎盘离体灌注数据与PBPK建模预测艾司西酞普兰、舍曲林和帕罗西汀在母亲及胎儿中的药物暴露Prediction of Maternal and Fetal Exposure to Escitalopram, Sertraline, and Paroxetine by Combining Human Ex Vivo Placenta Perfusion Data and Physiologically Based Pharmacokinetic ModelingPréta L.-H., Bouazza N., Foissac F., Froelicher L., Urien S., Buth V., Benaboud S., Tréluyer J.-M., Lui G. Clinical Pharmacokinetics. 2025 64:12 (1843-1854)ADC药物的上市后评估：基于模型荟萃分析和临床效用指数方法支持临床概念验证Postmarketing Assessment of Antibody–Drug Conjugates: Proof-of-Concept Using Model-Based Meta-Analysis and a Clinical Utility Index ApproachAsiimwe I.G., Chtiba N., Mouksassi S., Pillai G., Peter R.M., Yuen E., Pilla Reddy V. CPT: Pharmacometrics and Systems Pharmacology. 2025 14:11 (1810-1822)基于全身动态 PET 微剂量给药数据建立人体格列吡嗪全身药代动力学预测模型Modeling Whole-Body Dynamic PET Microdosing Data to Predict the Whole-Body Pharmacokinetics of Glyburide in HumansComin L., Marie S., Ursino M., Zohar S., Tournier N., Comets E. Clinical Pharmacokinetics. 2025 64:11 (1709-1722)家庭接触者中 SARS-CoV-2 检测的时间动态：首次阳性检测时间、症状出现时间与病毒载量峰值时间的差异Temporal Dynamics of SARS-CoV-2 Detection in Household Contacts: Divergences Between Time to First Positive Test, Symptom Onset, and Maximum Viral Load成人肺移植受者术后早期霉酚酸PopPK及贝叶斯评估Population pharmacokinetics of mycophenolic acid and Bayesian estimator in lung transplant adults recipients in the early post-transplant period抗中性粒细胞胞浆抗体（ANCA）相关性血管炎中利妥昔单抗诱导与维持治疗方案的比较：基于真实世界患者 ANCA 及丙种球蛋白水平的PK/PD分析Comparison of rituximab induction and maintenance regimens in anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis: PK/PD modelling of ANCA and gammaglobulin levels in real-world patientspH 值对德拉沙星抗大肠杆菌等基因株抗菌活性的影响：针对不同染色体和质粒介导的氟喹诺酮类耐药机制的研究Effect of pH on antimicrobial activity of delafloxacin against Escherichia coli isogenic strains carrying diverse chromosomal and plasmid-mediated fluoroquinolone resistance mechanisms头孢唑林在儿科心脏手术患者中作为预防用药的PopPK研究Population pharmacokinetics of cefazolin administered as prophylaxis in paediatric patients undergoing cardiac surgery比较 COVID-19 疫苗接种后未感染者和康复者随时间变化的中和抗体活性Comparing neutralizing antibody activity over time between naïve and convalesced COVID-19 vaccinated individualsPillis L., Caffrey R., Chen G., Dela M., Eldevik L., Liu H., McConnell J.P., Shabahang S., Varvel S.A. Scientific reports. 2025 15:1 (34800-)泼尼松龙在系统性红斑狼疮患者中的PopPK建模：暴露量与疾病活动度分析Population pharmacokinetic modelling of prednisolone in systemic lupus erythematosus patients: Analysis of exposure and disease activity利妥昔单抗治疗多发性硬化症临床预试验中的PK-PD建模：迈向个性化给药间隔的研究Pharmacokinetic–pharmacodynamic modelling in a clinical pilot study of rituximab in multiple sclerosis: Towards personalized dosing interval采用PopPK模型优化危重症难治性和超难治性癫痫持续状态患者的苯巴比妥给药方案Optimizing phenobarbital dosing in critically ill patients with refractory and superrefractory status epilepticus using a population pharmacokinetic model妊娠对囊性纤维化女性患者中 elexacaftor、tezacaftor 和 ivacaftor 药代动力学的影响Pregnancy-related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in cystic fibrosis womenMagnas P., Bouazza N., Foissac F., Froelicher-Bournaud L., Lui G., Carlier N., Da Silva J., Fesenbeckh J., Kanaan R., Honoré I., Martin C., Treluyer J., Tsatsaris V., Burgel P., Benaboud S. Journal of Cystic Fibrosis. 2025 24 Supplement 2 (S279a-S280)HIV 感染者中比克替拉韦的群体药代动力学：真实世界研究Population pharmacokinetics of bictegravir in real-world people with HIV社区获得性肺炎重症患者经持续输注高剂量头孢吡普后的肺内浓度研究Intrapulmonary concentrations of ceftobiprole high doses administered by continuous infusion in critically ill patients with community-acquired pneumonia血管紧张素 II/血管紧张素 I 比值作为接受依那普利治疗的健康成年受试者和心力衰竭儿童患者群体建模的一项新的药效学参数Angiotensin II/Angiotensin I Ratio as a New Pharmacodynamic Parameter for Population Modelling in Healthy Adults and Children with Heart Failure Treated with EnalaprilSteichert M., Cawello W., Burckhardt B.B., Suessenbach F.K., Laeer S. Pharmaceutics. 2025 17:10 Article Number 1345精确调整达托霉素给药剂量：3 种、2 种、1 种及 0 浓度取样方案比较Precision Daptomycin Dosing: Comparison of 3-, 2-, 1-, and 0-Concentration Sample StrategiesGiuliano S., Pai M.P., Angelini J., Flammini S., Martini L., Baraldo M., Tascini C. Pharmacotherapy. 2025 45:9 (540-546)针对接受肾脏替代治疗的危重患者的美罗培南和哌拉西林/他唑巴坦优化给药方案Meropenem and piperacillin/tazobactam optimised dosing regimens for critically ill patients receiving renal replacement therapyRoberts J.A., Ulldemolins M., Liu X., et al. Intensive Care Medicine. 2025 51:9 (1628-1640)基于PopPK模型优化利奈唑胺给药方案：针对疑似或确诊革兰阳性菌败血症的血液肿瘤患者Population Pharmacokinetic Model-Based Optimization of Linezolid Dosing in Hematooncological Patients With Suspected or Proven Gram-Positive SepsisZavřelová A., Merdita S., Žák P., Radocha J., Víšek B., Lánská M., Maláková J., Michálek P., Slanař O., Šíma M. Clinical and Translational Science. 2025 18:9 Article Number e70346IgG 编码 mRNA的多尺度机理药代动力学—毒代动力学（PK-TK）建模在基础研究到临床的应用：以抗 Claudin 18.2 抗体为例Bench to Bedside Modeling of mRNA Encoding IgG Using a Multiscale Mechanistic Pharmacokinetic-Toxicokinetic (PK-TK) Model: A Case Study With Anti-Claudin 18.2Desai D.A., Cristofoletti R. Clinical and Translational Science. 2025 18:9 Article Number e70356使用 SVG-101 群体药动学模型评估其合理的剂量选择Application of population pharmacokinetic modeling of SVG-101 to evaluate proper dose selectionMo K.H., Chae D., Jung Y.S., Jin B.H., Keum D.H., Choi M.K., Cha J.S., Park M.S., Kim C.O. Translational and Clinical Pharmacology. 2025 33:3 (156-167)2 型糖尿病患者代谢年龄和实际年龄vs碘海醇药代动力学的相关性Correlation of Metabolic Age & Calendar Age to Iohexol Pharmacokinetics in Patients With Type 2 DiabetesAlikhani R., Hooper L., Matvekas A., Waltje A., Rothberg A., Pai M. Clinical Pharmacology in Drug Development. 2025 14 Supplement 1 (6-7)通过群体PK/PD模拟评估替格瑞洛给药剂量中断及恢复治疗（含或不含负荷剂量）的效果Evaluation of the Effect of Ticagrelor Dose Interruption and Treatment Resumption with or without Bolus Doses Through Population PK/PD SimulationMatsui H., Pham L.T.T., Adane E. European Journal of Drug Metabolism and Pharmacokinetics. 2025 50:5 (409-418)缬更昔洛韦预防巨细胞病毒感染的目标达成情况Target attainment of valganciclovir prophylaxis against cytomegalovirusWenker S., Van Hasselt C., Moes D., Märtson A. British Journal of Clinical Pharmacology. 2025 91 Supplement 1 (8-9)胺碘酮对心脏手术后患者中阿哌沙班药物暴露的影响：一项群体药代动力学研究Effect of Amiodarone on Apixaban Exposure in Patients after Cardiac Surgery—A Population Pharmacokinetic StudyMorath B., Foerster K.I., Chiriac U., Zaradzki M., Hoppe-Tichy T., Schrey D., Burhenne J., Czock D., Karck M., Haefeli W.E., Wicha S.G. Clinical Pharmacokinetics. 2025 64:8 (1191-1201)非甾体抗炎药在山地超级越野跑者干血斑中的定量分析：PK模型对结果解读的贡献Quantitative analysis of nonsteroidal anti-inflammatory drugs in dried blood spot from mountain ultra-trail runners. Contribution of pharmacokinetic models for the interpretation of the resultsMashal M.S., Guitton J., Sallet P., Bourguignon L., Machon C. Drug Testing and Analysis. 2025 17:6 (825-833)比较不同胰岛素类型的疗效：健康受试者葡萄糖钳夹效应的PK-PD建模Comparing the Efficacy of Various Insulin Types: Pharmacokinetic and Pharmacodynamic Modeling of Glucose Clamp Effects in Healthy VolunteersChang Y.C., Jusko W.J. Journal of Clinical Pharmacology. 2025 65:8 (999-1010)评估多重插补算法对PK模型性能的影响：一项基于模拟的研究Assessing the Impact of Multiple Imputation Algorithms on Pharmacokinetic Model Performance: A Simulation-Based StudyDuflot T., Fayette L., Konecki C., Seurat J., Feliu C., Scala-Bertola J., Djerada Z.AAPS Journal. 2025 27:4 Article Number 77量化影响常染色体显性肾小管间质肾病进展速度和严重程度的临床及遗传因素Quantifying clinical and genetic factors influencing rate and severity of autosomal dominant tubulointerstitial kidney disease progressionRamesh S.S., Rogge M., Kidd K.O., Williams A.H., Yoon D.Y., Roignot J., Blakeslee K., Bleyer A.J., Kim S. Journal of Pharmacokinetics and Pharmacodynamics. 2025 52:4 Article Number 41甲氨蝶呤的治疗药物监测、PopPK建模及在儿童急性淋巴细胞白血病患者高剂量给药的外部验证Therapeutic Drug Monitoring, Population Pharmacokinetics Models, and External Validation of High-Dose Methotrexate in Pediatric Acute Lymphoblastic LeukemiaMaximova N., Calabrò P.F., Cangialosi A., Di Paolo A. Chemotherapy. 2025 70:3 (109-118)危重症儿童尿液生物标志物与头孢吡肟治疗目标达成的关联研究Urinary Biomarkers and Attainment of Cefepime Therapeutic Targets in Critically Ill ChildrenDownes K.J., Sharova A., Amajor V., Gianchetti L., Himebauch A.S., Fitzgerald J.C., Zuppa A.F. Pediatric Infectious Disease Journal. 2025 44:8 (749-754)Andalusian马体内静脉注射对乙酰氨基酚及其代谢物后的群体药代动力学和临床评估研究Population pharmacokinetics and clinical evaluation of intravenous acetaminophen and its metabolites in Andalusian horses预测妊娠期间他克莫司的游离浓度Predicting Unbound Tacrolimus Concentrations during PregnancyLeino A., Wilson N.K., Kataria A.D., Hebert M.F. American Journal of Transplantation. 2025 25:8 Supplement 1 (S204-)AEROfen：一项 I 期临床、开放标签、随机交叉研究方案，通过PK模型评估健康受试者中雾化芬太尼的给药效率—比较面部与鼻内给药途径AEROfen: protocol for a phase I, open-label, randomised crossover study evaluating the efficiency of nebulised fentanyl in healthy volunteers - comparing facial versus intranasal administration via pharmacometric modellingFollet C., Dumont A., Roussel M., Gillibert A., Boedard C., Quillard M., Ruault S., Vallin F., Donnadieu N., Nunes Ferreira D., Pereira T., Joly L.-M., Lvovschi V., Duflot T. BMJ Open. 2025 15:7 Article Number e091125超越线性模型的浓度- QT 分析Beyond the linear model in concentration‑QT analysisGéraldine Cellière, Andreas Krause, Guillaume Bonnefois, Jonathan Chauvin. Journal of Pharmacokinetics and Pharmacodynamics. (2025) 52:311型干扰素介导的系统性红斑狼疮炎症的综合机制模型An Integrative Mechanistic Model of Type 1 IFN-Mediated Inflammation in Systemic Lupus ErythematosusVolkova A., Sokolov V., Tettamanti F., Verma M., Ugolkov Y., Peskov K., Tang W., Kimko H. CPT: Pharmacometrics and Systems Pharmacology. 2025 14:7 (1225-1235)PK/PD建模与蒙特卡洛模拟预测接受（缬）更昔洛韦治疗的儿科移植受者的巨细胞病毒载量Pharmacokinetic/Pharmacodynamic Modelling and Monte Carlo Simulations to Predict Cytomegalovirus Viral Load in Pediatric Transplant Recipients Treated with (val) GanciclovirKoloskoff K., Franck B., Benito S., Welzel J., Autmizguine J., Theoret Y., Briand A., Ovetchkine P., Woillard J.-B. Clinical Pharmacokinetics. 2025 64:7 (1061-1069)沙特阿拉伯儿童肝移植术后早期他克莫司药代动力学的一项回顾性分析A retrospective analysis of tacrolimus pharmacokinetic in Saudi paediatric patients in early post-liver transplantation periodAlghanem A., Joharji H., Garaween N., Alenazi H., Alsaleh N.A., Broering D., Alshagrani M., Alhassan F., Albassam A.A., Alsultan A., Alsmari A. 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CPT: Pharmacometrics and Systems Pharmacology. 2025 14:6 (1065-1076)基于治疗药物监测, 考察阿米卡星在教学医院患者中的群体药代动力学Therapeutic Drug Monitoring-Based Population Pharmacokinetics of Amikacin in Patients at a Teaching HospitalSteffens N.A., Zimmermann E.S., Azeredo F.J., Linden R., Finatto L.J., Hahn R.Z., Schwarzbold A.V., Pacheco L.S., Brucker N. Antibiotics. 2025 14:6 Article Number 531辅助靶点的药效学评估：低分子量 PBP7/8 对β-内酰胺类药物抗碳青霉烯耐药鲍曼不动杆菌活性的影响Pharmacodynamic Evaluation of Adjuvant Targets: Low Molecular Weight PBP7/8 Effects on β-Lactam Activity Against Carbapenem-Resistant Acinetobacter BaumanniiHo B.M., Jin J., Sanborn J.T., Nguyen T.D., Singh N., Cheng C., Nasief N.N., Carlino-MacDonald U., Tsuji B.T., Zhao Y., Chen L., Moya B., Russo T.A., Smith N.M.Pharmaceuticals. 2025 18:6 Article Number 918WS02.06 Elexacaftor/tezacaftor/ivacaftor在 2 至 5 岁儿童中的药代动力学及行为障碍发生情况WS02.06Elexacaftor/tezacaftor/ivacaftor pharmacokinetics and occurrence of behavioural disorders in children aged 2 to 5 years oldTruong N.H., Benaboud S., Chouchana L., Bonnel A.-S., Barboura M., Bouazza N., Gautier S., Rouillon S., Froelicher-Bournaud L., Bihouee T., Bui S., Treluyer J.-M., Gaudelus I.S., Foissac F., Modul-CF research group. Journal of Cystic Fibrosis. 2025 24 Supplement 1 (S5-)老年群体中皮下注射与静脉注射头孢曲松的药代动力学：PhASAge 研究Pharmacokinetics of Subcutaneous and Intravenous Ceftriaxone in an Older Population: The PhASAge StudyRoubaud-Baudron C., Fauchon H., Stanke-Labesque F.,et al. Open Forum Infectious Diseases. 2025 12:6 Article Number ofaf313感染后期植入的 SIV 前病毒存在于寿命较短的 CD4+ T 细胞中SIV proviruses seeded later in infection are harbored in short-lived CD4+ T cells一箭多雕：用于多适应症的肠溶型霉酚酸钠的PopPK模型及贝叶斯估算Killing several birds with one stone: A multi-indication population pharmacokinetic model and Bayesian estimator for enteric-coated mycophenolate sodiumFromage Y., Sayadi H., Koloskoff K., Marquet P., Labriffe M., Monchaud C., Woillard J.-B. British Journal of Clinical Pharmacology. 2025 91:5 (1396-1408)通过PopPL分析优化 1-3 级肥胖患者静脉注射利多卡因的给药方案Dosing optimisation of intravenous lidocaine in patients with class 1–3 obesity by population pharmacokinetic analysisTognolini A.R., Liu X., Pandey S., Roberts J.A., Wallis S.C., Jackson D., Eley V.A. Anaesthesia. 2025 80:5 (511-521)用于考察阿替洛尔与美托洛尔在营养不良大鼠体内吸收的迷你PBPK模型Minimal Physiologically-Based Pharmacokinetic Modeling of Atenolol and Metoprolol Absorption in Malnourished RatsKir F., Sahin S., Jusko W.J. European Journal of Drug Metabolism and Pharmacokinetics. 2025 50:3 (251-263) Article Number e115Elexacaftor/Tezacaftor/Ivacaftor在囊性纤维化儿科患者中的群体药代动力学研究Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic FibrosisTruong N.H., Benaboud S., Bouazza N., Barboura M.,et al. Clinical and Translational Science. 2025 18:5 Article Number e70245比较妊娠妇女在惊厥前期使用 75 mg与 150 mg阿司匹林的效果Comparing 75mg vs 150mg aspirin in pregnancy women at risk of preeclampsiaVinogradov R., Kavanagh O.N., Kamali F., Robson S. American Journal of Obstetrics and Gynecology. 2025 232:5 (e189-e190)克林霉素与利福平联合用药的剂量及给药途径Dosing and route of administration of clindamycin given in combination with rifampicinMagréault S., Berrah R., Kerroumi Y., Mimram L., Salmon D., Goulenok T., de la Selle A., Lefort A., Marmor S., Helali N.H., Zeller V., Jullien V. Clinical Microbiology and Infection. 2025 31:5 (832-838)舒更葡糖或新斯的明逆转维库溴铵诱导犬神经肌肉阻滞的速度：随机临床试验与药理学建模Speed of reversal by sugammadex or neostigmine after vecuronium-induced neuromuscular block in dogs: Randomized clinical trial and pharmacological modelingMartin-Flores M., Lorenzutti A.M., Markmann A.D., Araos J., Campoy L. Veterinary Anaesthesia and Analgesia. 2025 52:3 (328-337)阿昔洛韦及其主要代谢产物 9-羧甲氧基甲基鸟嘌呤在早期肝移植受者中的群体药代动力学及伐昔洛韦的安全性评估Population pharmacokinetics of aciclovir and its major metabolite 9-carboxymethoxymethylguanine and safety profile of valaciclovir in early liver transplant recipientsKably B., Briard M., Francoz C., Roux O., Houhou N., Mackiewicz V., Peytavin G., Durand F., Lê M.P. Journal of Antimicrobial Chemotherapy. 2025 80:5 (1302-1308)一种用于治疗慢性乙型肝炎的衣壳蛋白装配调节剂作用的多尺度模型A multiscale model of the action of a capsid assembly modulator for the treatment of chronic hepatitis B左乙拉西坦药代动力学在真实世界中的患者间变异性Real-world interpatient variability in the pharmacokinetics of levetiracetamChykharivska A., Kagan L., Wagner M., Brunetti L. Fundamental and Clinical Pharmacology. 2025 39:2 Article Number e13059接受连续性肾脏替代治疗的危重患者是否需要调整头孢洛林给药剂量？基于回顾性数据的头孢洛林PopPK模型及目标达成概率分析的剂量模拟Do Critically Ill Patients Undergoing Continuous Renal Replacement Therapy Require Ceftaroline Dosage Adjustments? Ceftaroline PopPK Model and Dosage Simulations with the Probability of Target Attainment Analysis Based on Retrospective DataAdamiszak A., Pietrzkiewicz K., Bartkowska-Śniatkowska A., Smuszkiewicz P., Kusza K., Grześkowiak E., Bienert A. Antibiotics. 2025 14:4 Article Number 347胶质母细胞瘤患者中帕米帕利总药量及游离药量的PopPK建模：药物处置机制解析与给药方案优化Population Pharmacokinetic Modeling of Total and Unbound Pamiparib in Glioblastoma Patients: Insights into Drug Disposition and Dosing OptimizationWickramasinghe C., Kim S., Jiang Y., Bao X., Yue Y., Jiang J., Hong A., Sanai N., Li J. Pharmaceutics. 2025 17:4 Article Number 524美罗培南在严重细菌感染患者中的群体药动学与基于模型的给药方案优化Meropenem population pharmacokinetics and model-based dosing optimisation in patients with serious bacterial infectionMurínová I., Švidrnoch M., Gucký T., Rezáč D., Hlaváč J., Slanar O., Šíma M. European Journal of Hospital Pharmacy. 2024 31:3 (253-258)万古霉素在越南儿科患者治疗药物监测中的PopPK建模及中点浓度便捷采样Population Pharmacokinetic Modeling and Convenient Sampling of Midpoint Concentration for Therapeutic Drug Monitoring of Vancomycin in Vietnamese Pediatric PatientsHai L.B., Kien P.C., Dua N.T., Hao N.T., Hung V.M., Hai N.T., Le J., Huong N.T.L. Pharmaceutical Sciences Asia. 2024 51:3 (241-249)影响万古霉素谷浓度的因素：沙特非重症监护患者的PopPK模型Factors Affecting Vancomycin Trough Concentration; a Population Pharmacokinetic Model in Non-Critical Care Saudi PatientsOrvacabtagene Autoleucel, 一种自体 BCMA 导向的CAR-T 细胞产品在治疗复发/难治性多发性骨髓瘤患者中的PopPK研究Population Pharmacokinetics of Orvacabtagene Autoleucel, an Autologous BCMA-Directed Chimeric Antigen Receptor T-cell Product, in Patients with Relapsed/Refractory Multiple MyelomaHu H., Li Y., Piasecki J., Hosseyni D., Yan Z., Liu X., Ogasawara K., Zhou S., Cheng Y. Clinical Cancer Research. 2025 31:6 (1163-1171)建模分析表明，奈玛特韦-利托那韦治疗后出现的 SARS-CoV-2 病毒反弹，是由靶细胞得以保留与病毒清除不彻底共同导致的Modeling suggests SARS-CoV-2 rebound after nirmatrelvir-ritonavir treatment is driven by target cell preservation coupled with incomplete viral clearancePhan T., Ribeiro R.M., Edelstein G.E., Boucau J., et al.Journal of Virology. 2025 99:3用于癌症药物PK-PD建模与模拟的多器官芯片Multiorgan-on-a-chip for cancer drug pharmacokinetics-pharmacodynamics (PK-PD) modeling and simulationsMohammed A.E., Kurucaovalı F., Okvur D.P. Journal of Pharmacokinetics and Pharmacodynamics. 2025 52:1 Article Number 1NX210c多肽在健康老年受试者中的安全性、耐受性及PK-PD关系研究：一项随机、安慰剂对照、双盲、多剂量递增试验Safety, Tolerability and Pharmacokinetic-Pharmacodynamic Relationship of NX210c Peptide in Healthy Elderly Volunteers: Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Dose Study卡泊芬净在接受体外膜氧合治疗的危重患者中的PopPK研究——ASAP ECMO 研究Population pharmacokinetics of caspofungin in critically ill patients receiving extracorporeal membrane oxygenation—an ASAP ECMO studyAbdul-Aziz M.H., Diehl A., Liu X., Cheng V., Corley A., Gilder E., Levkovich B., McGuinness S., Ordonez J., Parke R., Pellegrino V., Wallis S.C., Fraser J.F., Shekar K., Roberts J.A. Antimicrobial Agents and Chemotherapy. 2025 69:2肾功能不全患者中哌拉西林剂量调整：无论是否接受肾脏替代治疗, 均需增加达到目标暴露量Dosing of Piperacillin in Altered Renal Function with and without Renal Replacement Therapy for Required Increased ExpositionSurat G., Dohmann E., Scherf-Clavel O., Kurlbaum M., Hagel S. Open Forum Infectious Diseases2025 12 Supplement 1 (S798-)重症成人鲍曼不动杆菌感染患者口服米诺环素的PK模型Phamacokinetic Model of Oral Minocycline in Critically Ill Adult Patients with Acinetobacter baumannii InfectionKoumaki V., Athanassa Z., Valsami G., Papakyriakopoulou P., Saitani E.-M., Marques S., Manioudaki S., Sakagianni A., Dokoumatzidis A., Tsakris A. Open Forum Infectious Diseases. 2025 12 Supplement 1 (S801-)使用容积吸收微量采样法研究多器官功能障碍综合征危重患儿中的万古霉素群体药代动力学Vancomycin Population Pharmacokinetics Using Volumetric Absorptive Microsampling in Critically Ill Children with Multiple Organ Dysfunction SyndromeShiau J., Amajor V., Rhodes N.J., Zuppa A., Downes K.J., Takyi-Williams J., Wen B., Scheetz M.H. Open Forum Infectious Diseases. 2025 12 Supplement 1 (S788-S789)粒细胞集落刺激因子的群体PK-PD建模以优化 CD34+细胞采集的给药剂量与给药时机Population Pharmacokinetic–Pharmacodynamic Modeling of Granulocyte Colony-Stimulating Factor to Optimize Dosing and Timing for CD34+ Cell HarvestingXu Jiang, Jun Seok Cha, Byung Hak Jin, Choon Ok Kim, Dongwoo Chae. Clinical and Translational Science, 2025; 18:e70121基于PopPK模型，为接受血液透析的肥胖患者群体制定万古霉素负荷剂量个体化给药方案Vancomycin loading dose individualization in a population of obese patients undergoing haemodialysis based on population pharmacokinetic modelPolášková L., Hartinger J.M., Murínová I., Michálek P., Slanař O., Šíma M. Journal of Chemotherapy. 2025 37:2 (121-129)考虑个体间变异的低维神经常微分方程在 Monolix 和 NONMEM 中的实现Low-dimensional neural ordinary differential equations accounting for inter-individual variability implemented in Monolix and NONMEMBräm D.S., Steiert B., Pfister M., Steffens B., Koch G. CPT: Pharmacometrics and Systems Pharmacology. 2025 14:1 (5-16)瑞德西韦及其代谢物在健康受试者和肾功能不全患者中的药代动力学模拟Pharmacokinetic simulations for remdesivir and its metabolites in healthy subjects and patients with renal impairment群体PK/PD分析证实 SB16 与参比药物地舒单抗具有生物相似性Population pharmacokinetics/pharmacodynamics analysis confirming biosimilarity of SB16 to reference denosumab肠溶阿司匹林胶囊与片剂在健康受试者群体中的PK-PD研究Population Pharmacokinetic and Pharmacodynamic Modeling of Enteric-Coated Aspirin Capsule and Tablet Formulations in Healthy Subjects结核病治疗中剂量精准的重要性The importance of getting the dose right in the treatment of tuberculosis瑞舒伐他汀和依折麦布联合用药导致肠肝循环的群体PK-PD研究Population Pharmacokinetics and Pharmacodynamics with Enterohepatic Recirculation of Co-Medication of Rosuvastatin and Ezetimibe在美国未感染 HIV 的成人中单独使用抗HIV-1 单克隆抗体 PGT121.414.LS，及与VRC07-523LS 联合给药的安全性、耐受性、药代动力学及中和活性研究（项目编号HVTN 136/HPTN 092）：一项首次人体、开放标签、随机对照的 1 期临床试验Safety, tolerability, pharmacokinetics, and neutralisation activities of the anti-HIV-1 monoclonal antibody PGT121.414.LS administered alone and in combination with VRC07-523LS in adults without HIV in the USA (HVTN 136/HPTN 092): a first-in-human, open-label, randomised controlled phase 1 trialEdupuganti S., Hurt C.B., Stephenson K.E., Huang Y., et al.The Lancet HIV. 2025 12:1 (e13-e25)恩诺沙星在治疗肉鸡大肠杆菌病中的PK、PD预测指标及蒙特卡洛模拟研究Pharmacokinetics, pharmacodynamic efficacy prediction indices, and Monte Carlo simulations of enrofloxacin for the treatment of colibacillosis in broiler chickens卡铂在犬类中的PopPK建模及其临床剂量调整Population pharmacokinetics modelling for clinical dose adjustment of carboplatin in dogsBéguin J., Mahfoudhi S., Uzel M., Rostang A., Ibish C., Ferran A.A., Pelligand L., Hulin A., Kohlhauer M. BMC Veterinary Research. 2024 20:1 Article Number 575Development of a Population Pharmacokinetic Gabapentin Model Leveraging Therapeutic Drug Monitoring ConcentrationsAl-Zubaydi F., Wassef A., Kagan L., Brunetti L. Pharmaceutics. 2024 16:12 Article Number 1514阿莫西林对犬类假中间葡萄球菌PK-PD评价Pharmacokinetics and Pharmacodynamics Evaluation of Amoxicillin Against Staphylococcus pseudintermedius in DogsJeong J.-S., Kim J.-W., Kim J.-H., Kim C.-Y., Chung E.-H., Boo S.-Y., Lee S.-H., Ko J.-W., Kim T.-W. Pathogens. 2024 13:12 Article Number 1121膳食和制剂对耐药性发育性及癫痫性脑病患儿体内大麻二酚全身暴露的影响Impact of food and formulation on cannabidiol systemic exposure in children with drug-resistant developmental and epileptic encephalopathiesBrstilo L., Reyes G., Ibarra M., Cáceres Guido P., Bressan I., Marin M.N., Delaven S.F., Agostini S., Pérez Montilla C., López M.E., Cresta A., García Bournissen F., Schaiquevich P., Caraballo R. Epilepsia. 2024 65 Supplement 1 (492-)基于模型荟萃分析建立复方口服避孕药突破性出血模型Development of breakthrough bleeding model of combined-oral contraceptives utilizing model-based meta-analysisChen H., Chun D., Lingineni K., Guzy S., Cristofoletti R., Hoechel J., Jiao T., Cicali B., Vozmediano V., Schmidt S. CPT: Pharmacometrics and Systems Pharmacology. 2024 13:11 (2016-2025)静脉注射免疫球蛋白在治疗各类免疫系统疾病患者中的PopPK模型Population Pharmacokinetic Model of Intravenous Immunoglobulin in Patients Treated for Various Immune System DisordersLee J.L., Mohamed Shah N., Makmor-Bakry M., Islahudin F., Alias H., Mohd Saffian S. Clinical Therapeutics. 2024 46:12 (e25-e37)持续输注哌拉西林/他唑巴坦可优化择期盆腔摘除术患者术中抗生素暴露水平Continuous infusion of piperacillin/tazobactam optimizes intraoperative antibiotic exposure in patients undergoing elective pelvic exenteration surgeryJackson D., Ulldemolins M., Liu X., Harris C., Tognolini A., Wallis S.C., Sumi C., Parker S.L., Eley V., Roberts J.A. Antimicrobial Agents and Chemotherapy. 2024 68:12接受单倍体相合干细胞移植并采用移植后环磷酰胺/环孢素 A 为基础的移植物抗宿主病预防方案的患者中，霉酚酸暴露量降低—需加强剂量调整Lower Mycophenolic Acid Exposure in Patients Undergoing Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide/ Cyclosporine a-Based GvHD Prophylaxis - Need for Dose IntensificationPai A.A., Panetta J.C., Bijukumar N., Renji R.M., A M., Jayaraman A., Karumathil S., Korula A., Selvarajan S., Srivastava A., Abraham A., Mathews V., George B., Kulkarni U., Balasubramanian P. Blood. 2024 144 Supplement 1 (2150-)不同剂量的甲氨蝶呤在类风湿关节炎患者中的PK-PD建模与模拟Pharmacokinetic-pharmacodynamic modelling and simulation of methotrexate dosing in patients with rheumatoid arthritis研究丙泊酚作为单一药物对 2-12 岁儿童双频谱指数峰值效应时间的影响Understanding time to peak effect of propofol as sole agent on bispectral index in children aged 2–12 yearsGavel G., Koch G., Terrier A., Atkinson A., Pfister M., Erb T. Paediatric Anaesthesia. 2024 34:10 (1019-1028)替扎帕林（一种皮下注射普通肝素的替代药物）用于重度及终末期肾功能不全患者的回顾性单中心观察性研究Tinzaparin, an alternative to subcutaneous unfractionated heparin, in patients with severe and end-stage renal impairment: a retrospective observational single-center study麦考酚酸酯在印度狼疮性肾炎患者群体中的PopPK及有限采样策略研究Population Pharmacokinetics and Limited Sampling Strategy of Mycophenolate Mofetil for Indian Patients With Lupus NephritisKoloskoff K., Panwar R., Rathi M., Mathew S., Sharma A., Marquet P., Benito S., Woillard J.-B., Pattanaik S. Therapeutic Drug Monitoring. 2024 46:5 (567-574)通过建模与模拟确定儿科重症监护室中美罗培南的最佳给药策略Identifying optimal dosing strategies for meropenem in the paediatric intensive care unit through modelling and simulation给药剂量是否得当？重症儿童患者中头孢吡肟的PopPK研究Are We Dosing Correctly? Population Pharmacokinetics of Cefepime in Critically Ill Pediatric PatientsRolsma S., Blackman M., Beck C., Dbouk A., Morovia F., Glover F., Ess G., Dohler K., Bridges B., Choi L., Creech C.B. Journal of the Pediatric Infectious Diseases Society. 2024 13 Supplement 4 (S9-S10)评估高加索健康受试者中与体重相关的协变量对利福平PK的影响Assessment of body mass-related covariates for rifampicin pharmacokinetics in healthy Caucasian volunteersBilal M., Ullah S., Jaehde U., Trueck C., Zaremba D., Wachall B., Wargenau M., Scheidel B., Wiesen M.H.J., Gazzaz M., Chen C., Büsker S., Fuhr U., Taubert M., Dokos C. European Journal of Clinical Pharmacology. 2024 80:9 (1271-1283)采用非线性混合效应模型对肥胖血液透析患者庆大霉素个体药代动力学参数进行估算Individual pharmacokinetic parameter estimation of gentamicin in an obese hemodialysis patient using non-linear mixed effect modelLee H., Yoon S., Chung J.Y. Translational and Clinical Pharmacology. 2024 32:3 (150-158)利用定量药理学评估患者友好的伊维菌素 CHILD-IVITAB 在体重≥15 kg 和 <15 kg 儿童中的给药剂量Pharmacometrics to Evaluate Dosing of the Patient-Friendly Ivermectin CHILD-IVITAB in Children ≥ 15 kg and <15 kgGolhen K., Buettcher M., Huwyler J., van den Anker J., Gotta V., Dao K., Rothuizen L.E., Kobylinski K., Pfister M. Pharmaceutics. 2024 16:9 Article Number 1186Elexacaftor/tezacaftor/ivacaftor 药代动力学与 2 至 5 岁儿童行为障碍的发生情况的研究Elexacaftor/tezacaftor/ivacaftor pharmacokinetics and occurance of behavioral disorders in children aged 2 to 5Truong N., Benaboud S., Chouchana L., Bonnel A., Barboura M., Bouazza N., Gautier S., Rouillon S., Froelicher-Bournaud L., Bihouee T., Bui S., Treluyer J., Sermet-Gaudelus I., Foissac F. Journal of Cystic Fibrosis. 2024 23 Supplement 2 (S277-S278)重症患者需增加头孢哌酮负荷剂量：一项采用PK建模评估推荐负荷剂量的回顾性研究Loading Dose of Ceftazidime Needs to Be Increased in Critically Ill Patients: A Retrospective Study to Evaluate Recommended Loading Dose with Pharmacokinetic ModellingLaunay M., Ollier E., Kably B., Le Louedec F., Thiery G., Lanoiselée J., Perinel-Ragey S. Antibiotics. 2024 13:8 Article Number 756达沙替尼在健康受试者中的PopPK研究Population Pharmacokinetics of Dasatinib in Healthy SubjectsHassouneh W.B., Al-Ghazawi M.A., Saleh M.I., Najib N. Pharmaceuticals. 2024 17:6 Article Number 671WS15.03 Elexacaftor/tezacaftor/ivacaftor 在囊性纤维化儿科患者中的PopPK研究WS15.03 Elexacaftor/tezacaftor/ivacaftor population pharmacokinetics in paediatric patients with cystic fibrosisTruong N.H., Benaboud S., Bardin E., et al. Journal of Cystic Fibrosis. 2024 23 Supplement 1 (S28-)慢性髓性白血病患者体重指数与血浆中伊马替尼浓度的关系，并采用PK引导的给药策略减轻其临床影响Relationship of body mass index and plasma imatinib levels in CML patients and mitigation of clinical impact using PK-guided dosing strategyMaroselli P., Fanciullino R., Colle J., Farnault L., Rocher P., Venton G., Costello R., Ciccolini J. Journal of Clinical Oncology. 2024 42:16 Supplement延长纳武利尤单抗给药间隔：在未筛选癌症患者中开展基于模型的模拟Extending the dosing intervals of nivolumab: model-based simulations in unselected cancer patientsPuszkiel A., Bianconi G., Pasquiers B., Balakirouchenane D., Arrondeau J., Boudou-Rouquette P., Bretagne M.-C., Salem J.-E., Declèves X., Vidal M., Kramkimel N., Guegan S., Aractingi S., Huillard O., Alexandre J., Wislez M., Goldwasser F., Blanchet B. British Journal of Cancer. 2024 130:11 (1866-1874)RPEM：随机蒙特卡洛参数期望最大化算法RPEM: Randomized Monte Carlo parametric expectation maximization algorithmChen R., Schumitzky A., Kryshchenko A., Nieforth K., Tomashevskiy M., Hu S., Garreau R., Otalvaro J., Yamada W., Neely M.N. CPT: Pharmacometrics and Systems Pharmacology. 2024 13:5 (759-780)肺移植受者进行伊沙康唑治疗药物监测的理论依据The Rationale for Therapeutic Drug Monitoring of Isavuconazole in a Lung Transplant RecipientZajacova A., Sima M., Havlin J., Klapkova E., Lischke R., Slanar O., Dvorackova E. Journal of Heart and Lung Transplantation. 2024 43:4 Supplement (S644-S645)Lumacaftor/Ivacaftor 在囊性纤维化儿科患者中的PopPK：迈向个体化治疗的第一步Lumacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients with Cystic Fibrosis: A First Step Toward Personalized TherapyBouazza N., Urien S., Foissac F., Choupeaux L., et al. Clinical Pharmacokinetics. 2024 63:3 (333-342)赖诺普利在高血压的儿童和正常至轻度肾功能受损的青少年的PopPK研究Population pharmacokinetics of lisinopril in hypertensive children and adolescents with normal to mildly reduced kidney functionSandra L., Degraeuwe E., De Bruyne P., De Baere S., Croubels S., Van Bocxlaer J.F.P., Raes A., Vande Walle J., Gasthuys E., Vermeulen A. British Journal of Clinical Pharmacology. 2024 90:2 (504-515)基于模型的临床前试验心血管安全性评估与转化教程A tutorial for model-based evaluation and translation of cardiovascular safety in preclinical trialsKulesh V., Vasyutin I., Volkova A., Peskov K., Kimko H., Sokolov V., Alluri R. CPT: Pharmacometrics and Systems Pharmacology2024 13:1 (5-22)提高奥拉帕利的药物暴露量以优化不良反应管理Improving olaparib exposure to optimize adverse effects managementSterlé M., Puszkiel A., Burlot C., Pereira E., Bellesoeur A., De Percin S., Beinse G., Fumet J.-D., Favier L., Niogret J., Blanchet B., Royer B., Bengrine-Lefevre L., Schmitt A. Therapeutic Advances in Medical Oncology. 2024 16疑似急性肾移植排斥反应：甲基泼尼松龙治疗下他克莫司的药代动力学研究Suspicious of Acute Kidney Graft Rejection: Tacrolimus Pharmacokinetics Under Methylprednisolone TherapyBidu N.S., Mattoso R.J.C., Santos O.A.C.O., Alves I.A., Fernandes B.J.D., Couto R.D. Current Drug Research Reviews. 2024 16:3 (403-411)►►►往年MonolixSuite文献汇总返回搜狐，查看更多

100 项与依伐卡托/芦马卡托相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	依伐卡托/芦马卡托	R07AX30	-

研发状态

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适应症	国家/地区	公司	日期
囊性纤维化	美国	Vertex Pharmaceuticals, Inc.	2015-07-02

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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


NCT04235140 (CTgov)	临床3期	囊性纤维化	52	LUM/IVA	願製糧選廠鹹構艱廠簾 = 簾餘齋鬱網獵窪築顧鬱遞製糧廠齋廠選築範鹹 (觸膚網膚淵範鹹鬱鬱衊, 觸醖構築鬱顧鏇壓顧齋 ~ 選鹽壓淵蓋衊製憲廠簾) 更多	-	2024-09-19
ERS2022	N/A	囊性纤维化 F508del	-	LUM/IVA cohort (US)	-	-	2022-09-04
ERS2022	N/A	囊性纤维化 F508del	-	LUM/IVA cohort (UK)	-	-	2022-09-04
NCT03625466 (CTgov)	临床2期	囊性纤维化	51	Placebo (Part 1: Placebo)	膚鹽憲鏇齋製鹽製構鑰(窪簾範鬱艱積構艱製鏇) = 鬱淵積鑰鹽夢齋廠衊鏇觸醖衊製壓窪積鹹選糧 (鬱膚廠獵構鹹窪構艱遞, 6.1) 更多	-	2021-12-08
NCT03625466 (CTgov)	临床2期	囊性纤维化	51	LUM/IVA (Part 1: LUM/IVA)	膚鹽憲鏇齋製鹽製構鑰(窪簾範鬱艱積構艱製鏇) = 構範繭製選鑰夢淵糧網觸醖衊製壓窪積鹹選糧 (鬱膚廠獵構鹹窪構艱遞, 6.6) 更多	-	2021-12-08
ERS2021	N/A	囊性纤维化 F508del	-	LUM/IVA	窪築鹹網顧襯廠糧壓鹽(鏇遞構鏇選襯窪積顧壓): OR = 0.16 (95% CI, 0.07 ~ 0.38) 更多	-	2021-09-05
ERS2021	N/A	囊性纤维化 F508del	-	lumacaftor/ivacaftor (LUM/IVA)	窪築鹹網顧襯廠糧壓鹽(鏇遞構鏇選襯窪積顧壓): OR = 0.16 (95% CI, 0.07 ~ 0.38) 更多	-	2021-09-05
NCT03061331 (Pubmed \| J Cyst Fibros) 人工标引	临床2期	囊性纤维化 A455E-CFTR Mutation	20	Placebo+Ivacaftor+Lumacaftor	獵網壓繭艱憲衊構淵淵(網鏇獵鏇艱鑰鬱蓋範鬱) = 鹹鹹壓鬱鬱鏇鬱觸艱廠鹽積觸齋築繭夢顧餘製 (顧醖壓選醖壓鬱齋遞鑰 )	不佳	2021-09-01
NCT03061331 (Pubmed \| J Cyst Fibros) 人工标引	临床2期	囊性纤维化 A455E-CFTR Mutation	20	Placebo+Ivacaftor+Lumacaftor	獵網壓繭艱憲衊構淵淵(網鏇獵鏇艱鑰鬱蓋範鬱) = 範網衊簾選糧網餘憲艱鹽積觸齋築繭夢顧餘製 (顧醖壓選醖壓鬱齋遞鑰 )	不佳	2021-09-01
NCT03125395 (study 116; VX16-809-116, Pubmed \| Lancet Respir Med) 人工标引	临床3期	囊性纤维化 F508del mutation	57	Ivacaftor/Lumacaftor	顧製顧構憲廠衊憲製顧(襯蓋膚衊網簾蓋餘淵選) = 選構積製鬱壓夢鹹範鑰衊積艱選餘窪餘願積淵 (遞範醖壓築醖鏇遞鏇醖 ) 更多	积极	2021-09-01
NCT02807415 (Pubmed)	N/A	囊性纤维化	30	Lumacaftor-Ivacaftor	鹽鏇遞簾選膚淵鑰選獵(憲衊憲遞艱鑰觸糧憲鑰) = 夢鹹衊醖餘鏇願壓壓製餘鏇蓋膚蓋簾齋繭選選 (餘遞糧鏇壓廠獵觸製願, -938.7 ~ 290.4)	-	2021-02-18
ERS2020	N/A	囊性纤维化 Phe508del mutation	5	Lumacaftor/Ivacaftor therapy	蓋願觸蓋選網網憲膚夢(製壓簾膚遞鏇築醖憲製) = 遞壓構觸憲鏇艱廠構繭齋顧蓋觸醖構遞繭鹽獵 (醖製獵構襯廠窪憲製壓, 24.9 ~ 38.4)	-	2020-09-07
NCT03125395 (study 116; VX16-809-116 \| VX16-809-116, CTgov)	临床3期	囊性纤维化	57	LUM/IVA	廠壓餘壓觸衊鏇鏇糧築 = 鏇壓艱廠願鹽鹹簾衊觸繭願遞蓋鏇願壓獵衊鏇 (顧鑰網願構鏇醖淵憲蓋, 鏇淵構窪齋鏇醖顧醖獵 ~ 鑰顧願鹹鹽顧選糧憲鹽) 更多	-	2020-08-07
Pubmed \| Am J Respir Crit Care Med	N/A	囊性纤维化	845	Lumacaftor-ivacaftor	鹹觸鬱鬱廠廠範選鹽夢(壓壓顧鹹獵觸願醖顧齋) = 窪醖簾願鹽簾網襯鏇糧醖築齋構醖餘構構淵積 (築鏇鹽鑰齋範遞鑰衊鑰 ) 更多	积极	2020-01-15