Accelerating Pediatric Oncology Drug Development: Advances in Clinical Pharmacology, Trial Design, Non-Clinical Evidence, and Regulatory Science [0.03%]
儿科肿瘤药物加速研发：临床药理学、试验设计、非临床证据及监管科学研究进展
Ya-Feng Wen,Xiaoning Wang,Sarandeep Boyanapalli et al.
Ya-Feng Wen et al.
Pediatric oncology drug development remains uniquely challenging due to the rarity and biological heterogeneity of childhood cancers, ethical considerations in trial conduct, and the limited feasibility of large, randomized studies. Despite...
Population Pharmacokinetic Modeling for the Iminosugar Lucerastat Supports Dose Adaptation in Patients With Fabry Disease and Moderate to Severe Renal Function Impairment [0.03%]
基于人群的药物动力学模型指导iminosugar类化合物Lucerastat在肾功能中至重度受损的法布雷病患者中的剂量调整
Janneke M Brussee,Jasper Dingemanse,Dominik Lott et al.
Janneke M Brussee et al.
Lucerastat is an iminosugar with the potential to provide substrate reduction therapy for the treatment of Fabry disease (FD), an inherited X-linked lysosomal storage disorder. The aims of this study were to develop a population pharmacokin...
Clinical Trial
Journal of clinical pharmacology. 2026 Jul;66(7):e70247. DOI:10.1002/jcph.70247 2026
Evaluating the Impact of Enfortumab Vedotin Dose Modifications on Clinical Efficacy Using Tumor Growth Inhibition Modeling in Patients With Advanced Urothelial Cancer [0.03%]
利用肿瘤生长抑制模型评估恩诺单抗剂量调整对晚期尿路上皮癌患者临床疗效的影响
Vaishali L Chudasama,Gabriela Patilea-Vrana,Hong Mu et al.
Vaishali L Chudasama et al.
Enfortumab vedotin (EV) is approved for previously treated locally advanced or metastatic urothelial cancer (mUC), based on results from pivotal trials (phase 2, EV-201; phase 3, EV-301) evaluating EV monotherapy at 1.25 mg/kg 3Q4W (Days 1,...
Extrapolating Efficacy of Cariprazine to Pediatric Schizophrenia and Bipolar Mania Through Population Pharmacokinetic Analysis [0.03%]
通过人群药代动力学分析外推卡利拉嗪治疗儿科精神分裂症和双相躁狂的有效性
Shams Ismaeil,Lucia Siovitz,Rahul K Goyal et al.
Shams Ismaeil et al.
The US FDA recently accepted efficacy extrapolation from adult to pediatric patients for atypical antipsychotics in the treatment of schizophrenia (SCZ) and bipolar mania (BPM), considering the similarity in both disease and exposure-respon...
Population Pharmacokinetic Modeling and Simulation to Support the Regulatory Submission of the Two-Injection Start Regimen with Aripiprazole Once-Monthly Long-Acting Injectable Intramuscular Administration in Japanese Patients with Schizophrenia or Bipolar I Disorder [0.03%]
支持日本精神分裂症或I型双相情感障碍患者使用艾司西酞普兰长效肌肉注射给药双针启动方案的监管提交的群体药代动力学建模与模拟研究
Yumiko Yamasaki,Tomohiro Sasaki,Antal Martinecz et al.
Yumiko Yamasaki et al.
To improve medication adherence in Japanese patients with schizophrenia or bipolar I disorder, this population pharmacokinetic (popPK) modeling and simulation was conducted to support the regulatory submission of the two-injection start (TI...
External Evaluation of Population Pharmacokinetic Models for Factor VIII in Chinese Patients with Hemophilia A [0.03%]
中国人血友病A患者第八因子人口药代动力学模型的外部评价
Jinxia Lu,Zipeng Wei,Baohua Xu et al.
Jinxia Lu et al.
Although numerous population pharmacokinetic (PopPK) models related to factor VIII (FVIII) replacement therapy have been published, the vast majority have not undergone external validation. This study aims to externally validate existing Po...
How to Replace the TQT Study-The Use of Concentration-QTc Modeling to Exclude a Small Effect of a Novel Drug on QT Interval: Historical Perspective and Implementation [0.03%]
如何替代TQT研究——应用浓度-QTc建模排除新药对QT间期的微小影响：历史回顾与实施方案探讨
Borje Darpo,Georg Ferber,Seth C Hopkins et al.
Borje Darpo et al.
The initial ICH E14 guidance described the thorough QT/QTc (TQT) study with the purpose of evaluating whether a new drug has effects on the QTc interval. Following its adoption, concentration-QTc (C-QTc) modeling was applied to data from TQ...
Genome Sequencing Enhances Precision and Clinical Utility of Pharmacogenetic Data Compared to Arrays [0.03%]
基因组测序技术相比基因芯片技术能够提高药物基因分型的精准性和临床应用性
Ibrahim Numanagić,Morgan Similuk,Tristan M Sissung et al.
Ibrahim Numanagić et al.
While pharmacogenetic testing has traditionally relied on array-based genotyping platforms, these methods are limited by incomplete variant coverage and inability to detect novel alleles. We hypothesize that the performance of genome sequen...
Comparative Study
Journal of clinical pharmacology. 2026 Jul;66(7):e70235. DOI:10.1002/jcph.70235 2026
FDA Gene Therapy Approvals (1998-2025): Current Status, Regulatory Evolution, and Future Directions [0.03%]
美国 FDA 基因治疗批准概览（1998年-2025年）：现状、法规演变和未来方向
Charles Oo,Xiaofa Qin,Sherwin Kb Sy
Charles Oo
FDA approvals of gene therapies began slowly and were concentrated within a few early modalities, but recent years have seen a marked acceleration across RNA-based agents, viral and non-viral in vivo platforms, and ex vivo genetically modif...
Human Disposition, Metabolism, and Excretion of Sevasemten (EDG-5506), a Selective Modulator of Fast Myosin in Healthy Volunteers [0.03%]
在健康志愿者中评估新型快速肌球蛋白选择性调节剂sevasertin（EDG-5506）的人体暴露、代谢和排泄研究
Molly Madden,Marc Evanchik,Jonathan Lane et al.
Molly Madden et al.
Sevasemten (EDG-5506) is a novel, orally bioavailable, investigational small molecule designed to selectively modulate type II fast skeletal myosin with the goal of protecting dystrophic muscle from contraction-induced injury. Sevasemten is...
Clinical Trial
Journal of clinical pharmacology. 2026 Jul;66(7):e70234. DOI:10.1002/jcph.70234 2026