An Open-label, Phase I/II Multicenter Clinical Trial of NECVAX-NEO1 as add-on to First-line Neoadjuvant Anti-PD-1 Monoclonal Antibody Therapy in Patients With Triple-negative Breast Cancer

Phase I/II, multicenter, open-label, single-arm trial in triple-negative breast cancer patients under first-line neoadjuvant therapy with approved standard of care anti-PD-1 monoclonal antibody (PD-1 inhibitor), epirubicin/cyclophosphamide chemotherapy, and nab-paclitaxel therapy. NECVAX-NEO1 treatment in addition to standard of care anti-PD1 monoclonal antibody therapy can be prolonged after breast cancer surgery for another 24 weeks, according to the investigator's decision taking into consideration the study patient's health status.

开始日期2024-11-20

申办/合作机构

NEC Corp.

[+1]

NCT06631079

/ Recruiting临床1/2期

An Open-label, Phase I/II Multicenter Clinical Trial of NECVAX-NEO1 in Addition to Anti-PD-1 or Anti-PD-L1 Monoclonal Antibody Therapy in Patients With Solid Tumors

Phase I/II multicenter, open-label, single-arm trial in patients to evaluate the safety and effect of NECVAX-NEO1 administered in combination with PD-1/PD-L1 mABs in patients with solid tumors. Patients with solid tumors who will be treated with approved standard of care (SoC) PD-1 or PD-L1 monoclonal antibody inhibitor therapy, and who after starting treatment with PD-1/PD-L1 inhibitor reached either Stable Disease (SD) or Partial Response (PR) (Cohort 1) or PD (Cohort 2) according to RECIST 1.1 and iRECIST assessed at the Baseline visit may be enrolled in the study

开始日期2024-10-07

申办/合作机构

NEC Corp.

[+1]

JPRN-UMIN000054560

/ SuspendedN/A

Evaluation of effectivity of accompanying type intervention by using FonesVisuas, the future disease risk prediction service - Evaluation of effectivity of accompanying type intervention by using FonesVisuas, the future disease risk prediction service

开始日期2024-07-01

申办/合作机构

NEC Corp.

100 项与 NEC Corp. 相关的临床结果

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0 项与 NEC Corp. 相关的专利（医药）

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项与 NEC Corp. 相关的文献（医药）

2025-08-01·COMPUTER METHODS AND PROGRAMS IN BIOMEDICINE

Development of patient-specific apparent blood viscosity predictive models for computational fluid dynamics analysis of intracranial aneurysms with machine learning approaches

Article

作者: Yamamoto, Makoto ; Fujimura, Soichiro ; Ishibashi, Toshihiro ; Suzuki, Tomoaki ; Hataoka, Shunsuke ; Aoki, Ken ; Yamamoto, Hideki ; Suzuki, Takashi ; Takao, Hiroyuki ; Kodama, Tomonobu ; Murayama, Yuichi

BACKGROUND AND OBJECTIVES:

A model to predict patient-specific apparent viscosity as a computational condition in computational fluid dynamics (CFD) analysis, which is used in research on intracranial aneurysms, is important. The purpose of this study was to develop a model to predict patient-specific apparent viscosity from clinical blood test results.

METHODS:

The data were from 15 patients with intracranial aneurysms in whom blood viscosity and density were measured and blood tests were performed on the same day. The dataset was divided into two, a training dataset and a test dataset at a ratio of 4:1. The training dataset was used in constructing regression models with shear rate and 12 blood test items (the flexible model) or hematocrit (the simple model) as input, and the measured apparent viscosity as output. CFD analysis was implemented with and without coil geometries, and the viscosity models were evaluated.

RESULTS:

The root mean squared error (RMSE) of viscosity predicted with the flexible model and the simple model was 0.136 mPa·s and 0.226 mPa·s, respectively. The RMSE of time-averaged and space-averaged velocity and time-averaged and space-averaged wall shear stress computed in CFD analysis were <0.01 m/s and <0.21 Pa, respectively.

CONCLUSIONS:

Regression models to predict patient-specific apparent blood viscosity from shear rate and blood test items were constructed with machine learning. There is a possibility that, using this predictive model, patient-specific blood apparent viscosity can be predicted with high accuracy from the blood test results of individual patients.

Scientific Reports

Factors associated with pressure ulcers among middle-old and oldest-old inpatients in an acute care hospital

Article

作者: Kodama, Kana ; Kobayashi, Kaori ; Nakagawa, Rie ; Kikuchi, Masataka ; Matsumura, Yasushi ; Murata, Taizo ; Abe, Kaori ; Itoh, Sakiko ; Takeda, Toshihiro ; Yokota, Shinichiroh ; Nabetani, Yoshiko ; Tanaka, Sumie ; Yotsu, Rie R

With the aging population, the majority of patients in acute care hospitals in high-income countries are older adults. To identify the factors associated with pressure ulcers among patients aged ≥ 75 years in an acute care hospital,we conducted a retrospective study using individual-level secondary data obtained from patient medical and nursing records. The study population was patients aged ≥ 75 years who were admitted to the hospital between March 2016 and February 2021. We fitted multivariable logistic regression models. A total of 15,258 were included in the analysis, and 513 (3.4%) patients were with pressure ulcers. The factors associated with pressure ulcers were Body Mass Index (adjusted OR = 0.96, 95% CI: 0.93-0.98); albumin level (0.51, 0.42-0.63); cardiovascular disease (2.10, 1.57-2.82); infection (1.91, 1.07-3.42); bedridden (1.85, 1.01-3.39); using bed when moving (0.45, 0.25-0.84); fecal or urine incontinence (2.44, 1.46-4.06); admission to the emergency medicine (1.71, 1.06-2.74); length of stay (1.02, 1.01-1.02); and tube feeding (2.02, 1.44-2.82). Low nutritional status, low activity levels, incontinence, cardiovascular disease, infection, admission to the emergency medicine, and more extended hospital stay were significantly associated with pressure ulcers. Therefore, nurses in acute care should carefully monitor the patients aged ≥ 75 years with these factors.

2025-06-01·CLINICAL BIOMECHANICS

Measurement of gait by an in-shoe motion sensor system: feasibility and potential application in healthy adults and patients with orthopedic diseases

Article

作者: Etoh, Norihito ; Takahashi, Hiroshi ; Asakura, Keiko ; Mori, Sachie ; Yoshida, Aya ; Nakano, Hiroaki ; Furukawa, Azusa ; Fukutake, Katsunori ; Aoki, Hideyuki ; Nishiwaki, Yuji ; Orito, Eisuke

BACKGROUND:

Gait varies in accordance with several factors, including age, gender, and the presence of disease. A recently developed system allows the convenient measurement of gait using in-shoe motion sensors and a smartphone application. Here, this system was used to measure seven gait parameters in 40 healthy adults and 45 patients with orthopedic disorders.

METHODS:

For the healthy volunteers, gait monitoring was performed for one month. This monitoring was also performed at home before and one year after surgery in patients with knee or spine disorders, and at hospital at four time points before and after surgery to monitor changes in gait. The relationship between participant characteristics and gait was assessed among the healthy volunteers, and change of gait in the perioperative period was described among the patients.

FINDINGS:

In the healthy volunteers, being in one's 80s was significantly related with worse gait conditions, such as slower walking speed and shorter stride length, than being in one's 40s. Also, higher height was associated with longer stride length. In the patients with knee disorders, many gait parameters worsened from before to one month after surgery, and then tended to improve up to one year post-surgery. In contrast, patients with spinal disorders showed improvement even at one month post-surgery, with continued improvement up to three months post-surgery.

INTERPRETATION:

Measurement in daily life was feasible, and thought to accurately reflect the gait of each participant. Further studies are needed to investigate the relationship between the gait parameters and health outcomes.

项与 NEC Corp. 相关的新闻（医药）

2025-06-27

·研发客

日本临床试验申报制度解析：分类、流程与应对

撰文｜东内祥浩（WaRA Consulting）翻译｜文英淑（泰格医药日本公司副总裁）中文版翻译负责人｜医药研发达人主编高野哲臣（t2T Healthcare股份公司总裁兼首席执行官）2025年6月27日出版，医药研发达人中文版“出海日本”第12期，系列B《日本的监管制度及其实际情况》第五篇 // · 日本PMDA对临床试验申报分类严格，并明确不同类型试验的申报时间要求，如需30天调查的临床试验需提前至少30天申报，确保安全合规。· 资料明确了临床试验变更和终止时的申报规范，要求变更前提交变更申报书，终止时立即申报，保障临床试验透明度和可追溯性，便于监管机构及时掌握动态。· 30天调查的临床试验流程复杂，要求申办者快速回应PMDA问询；而14天规则适用试验流程简便，建议提前咨询，以提高申报效率。本连载的系列B将介绍日本药品监管制度的基本信息。这一期将介绍在日本的临床试验申报制度。计划实施临床试验的个人、组织或者研究机构（以下统称申办者）在日本开展涉及如下试验药物的临床试验时，必须向PMDA申报临床试验。“试验药物”是指作为临床试验对象的药物，该临床试验的结果用于支持该药物在日本的生产销售注册申请。另一方面，临床试验中用于评价试验药物有效性和安全性的对照药物、联合用药、补救用药、试验药物给药前药物等称为临床试验的使用药物。涉及以下试验药物的临床试验：① 与已批准药物活性成分不同的药物② 与已批准药物活性成分相同但给药途径不同的药物③ 与已批准药物的活性成分比例、功效、作用、用法或用量不同的药物④ 与已批准药物活性成分相同但再审查尚未到期的药物⑤ 生物制剂⑥ 利用重组基因技术生产的药物1. 临床试验的申报时间申报时间原则上取决于申报类型。① 临床试验申报书（用于临床试验申报）(a)如果所申报的临床试验属于需进行30天调查*的范畴，申办者应在计划与研究机构签订合同的至少30天前申报临床试验。临床试验合同必须在申报30天后签订。需进行30天调查*的试验药物：在日本首次用于人体的以下试验（无论该药物是否有海外使用经验）。i) 与已批准药物等活性成分不同的药物。ii) 与已批准药物等活性成分相同但给药途径不同的药物。iii)与已批准药物等活性成分比例不同的药物。(b)除上述以外的临床试验，申办者应在计划与研究机构签订合同的至少两周前申报临床试验。但是，如果所申报的临床试验涉及I期试验等，则建议提前与PMDA协商申报时间。笔者精选信息630天调查与14天规则需要进行30天调查的临床试验，临床试验申办者应在计划与研究机构签订合同的至少31天前申报临床试验，而其他临床试验，需要在计划与研究机构签订合同的至少两周前申报临床试验。虽然两种临床试验的申报时间在PMDA发布的相关通知里描述略有不同，但实际流程却大相径庭。申报临床试验的目的首先是确保“针对日本人”的临床试验的安全性，因此，即使有海外人体试验的临床试验数据，在日本首次进行人体试验的临床试验时，PMDA会根据非临床研究结果和临床试验进展情况，谨慎确认试验药物的安全性。针对需要进行30天调查的临床试验，在申办者申报临床试验大约一周后，PMDA将提出针对申报资料里所附的试验方案、知情同意书等的问询事项。申办者需要对此作出回应，大多数情况下，需要修改方案和/或知情同意书。PMDA会基于申办者的回应和修改后的文件开展调查，并在申办者申报临床试验的25天后将调查结果上报到厚生劳动省（MHLW）。MHLW将确认上报的内容，完成30天调查。换句话说，从PMDA发出问询到提交回应和修改的文件只有18天（约14个工作日）的时间，根据问询的内容，如果涉及方案修订等耗时很长的工作时，回复的截止日期迫在眉睫。特别是，如果申办者的总部位于海外，时间安排会变得更加紧张。在这种情况下，即使申办者提出延期与研究机构签订合同的时间，PMDA也不会因此接受延长调查期的请求。30天的调查期需要严格遵守，因此如果申办者无法在该期限内作出回应，则必须撤回所申报的临床试验，修改必要的部分后重新申报，并接受新一轮30天的调查。这可能会推迟临床试验的启动时间。而对于不受30天调查约束的临床试验，适用14天规则。由于这类试验并非是在日本首次进行人体试验，因此在大多数情况下，PMDA不会发出问询。如果14天后未收到PMDA的反馈，则申办者可以与研究机构签订合同。为了减少30天调查对启动临床试验的阻碍， PMDA在其网站（网站信息请参阅参考文献）上发布了与30天调查相关的问询确认清单（针对抗癌药物和生物制品质量部分，均为日语）以及首次提交临床试验申报书时需要考虑的非临床安全事项（早期考量）《原文：Points to Consider for nonclinical safety matters when submitting the initial clinical trial notification (Early Consideration)》。然而，正如笔者在上一篇文章中提到的，建议申办者在首次申报临床试验之前，先与PMDA咨询，得到相关建议后，再准备相应的文件用于申报临床试验。② 临床试验变更申报书（用于临床试验变更申报）(a)临床试验申报书的内容发生变更时，原则上必须在每次变更前提交临床试验变更申报书。(b)在临床试验变更申报书里增加需接受30天调查的试验药物时，必须在开展变更后的临床试验（按照增加该试验药物的方案）的至少30天前进行申报。(c)在临床试验变更申报书增加不需要30天调查的试验药物或在日本尚未积累足够安全性信息的临床试验的使用药物时，必须在开展变更后的临床试验（按照增加该试验药物或者临床试验使用药物的方案）的约两周前进行申报。此外，变更目的或目标疾病时，必须在开展变更后的临床试验（按照变更目的或目标疾病的方案）的约两周前进行申报。(d)以下事项可在变更发生后约6个月内一并申报：・生产或销售机构名称、地点、企业代码发生变化，但实际情况没有改变・因《日本药典》修订而导致的通用名变更，以及JAN (Japanese Accepted Names for Pharmaceuticals,日本药品通用名称）的确定等导致的成分和含量的变更，但实际情况没有改变・仅变更进口国生产商的名称，以及进口国销售名称的变更等实际情况没有改变生产方法的变更・因与最早签订临床试验合同的研究机构签订合同日期的变更，导致的临床试验实施期间发生变更的微小变更（请注意，如果计划推迟研究机构的最终观察结束预计日期，则必须事先申报）・删除临床试验协调研究者及临床试验协调委员会成员，变更临床试验协调研究者及临床试验协调委员会成员的姓名、所属机构、所属部门・负责临床试验申办及管理相关全部或部分工作的受托方【合同研究组织 (CRO) 】的名称、地址及委托工作范围的变更及增删・临床试验申报人（见下文“4临床试验申报人”）的名称（公司名称变更及代表人变更，但实际情况未发生改变）、地址及委托工作范围的变更・临床试验申报人的具体申报人员的姓名、所属部门、电话号码、传真号码或电子邮件地址的变更及增删・外国生产企业名称（公司名称变更及代表人变更，但实际情况未发生改变）及地址的变更・未签订合同的研究机构的删除，研究机构名称、诊疗科室、所在地及总机电话号码的变更・主要研究者姓名的变更・因删除研究机构而删除主要研究者・接受研究机构委托开展部分临床试验相关工作的受托方【例如临床试验现场管理组织 (SMO) 】的名称、地址及委托工作范围的变更及增删・伦理委员会设立方的名称和所在地的变更及增删(e)临床试验申报人变更导致实际情况发生变化的，需要重新申报，而非提交变更申报书。(f)以下事项无需按照临床试验变更申报，可以按照临床试验完成或临床试验提前终止一并申报。・临床试验的使用药物计划发放（采购）的数量（计划供应给研究机构的数量）及受试者预计人数的变更・次级研究者的姓名变更及增删③临床试验提前终止申报书（用于临床试验提前终止申报）针对每个临床试验申报书，只要临床试验提前终止，应立即申报。④临床试验完成申报书（用于临床试验完成申报）针对每个临床试验申报书，收到所有参与研究机构的临床试验完成通知、并回收所有试验用药品后，应立即申报2. 申报临床试验时应附的资料原则上应根据申报类型提交如下文件作为附件。① 临床试验申报，除提供临床试验申报书以外，提交如下文件。· 说明认为该临床试验申办在科学上正当性的理由的文件· 临床试验方案· 用于知情同意的说明文件及同意书· 病例报告表样本· 最新研究者手册· 质量相关文件（如有必要，包括与DNA反应性（致突变性）杂质评估和管理相关的文件，或与使用已建立的细胞系生产的蛋白质药物质量相关的文件等）② 临床试验变更申报，除提供临床试验变更申报书以外，提交如下文件。根据情况提供与变更相关的文件。③ 临床试验提前终止申报，除提供临床试验提前终止申报书以外，提交如下文件。根据情况提供与提前终止原因相关的文件（包括提前终止前所实施的病例信息）。3. 研发终止申报书如果决定终止已申报的临床试验中试验药物的研发，申办者必须在决策后立即向PMDA提交研发终止申报书。此外，申办者必须具体说明终止研发的理由。请注意，申办者自首次申报临床试验之日起就必须履行报告安全性信息的义务，直至获得批准或提交研发终止申报书为止。因此，务必牢记，即使在一个临床试验结束后根据其结果讨论是否继续或终止开发之时，仍需报告安全性信息。4. 关于临床试验申报人临床试验的申报人是申办者或国内临床试验管理人（ICCC：In-Country Clinical Caretaker）。在日本，如果有意在日本开展临床试验的申办者在日本没有成立法人，则必须指定国内临床试验管理人（ICCC）代表申办者申办临床试验。通常，国内临床试验管理人对所开展的临床试验负有责任，并与业务受托方CRO共同开展临床试验。该临床试验的最终责任由外国的申办者承担。参考文献·30天调查问询事项确认清单https://www.pmda.go.jp/review-services/trials/0024.html·Points to Consider for nonclinical safety matters when submitting the initial clinical trial notification (Early Consideration)https://www.pmda.go.jp/files/000274660.pdf预告下期（第13期）是由高野哲臣先生撰写的系列A《日本的临床试验和药品市场》第六篇《日本的临床试验费用计算方法》，预计将于2025年7月上旬出版。中文版第12期作者简介东内祥浩先生（Yoshihiro Higashiuchi）东内祥浩先生是一位拥有20年经验的资深监管事务顾问、监管科学家，负责领导监管活动，包括与药品医疗器械综合机构（PMDA）的咨询、新药申请（NDA）提交和新分子实体（NME）、新适应症/产品线扩展（NILEX）的监管批准，其中包括5个NME和3个NILEX的监管批准。通过13年（自2008年起）组织策划日本药物信息协会（DIA）监管事务培训课程的活动，东内祥浩与PMDA的审评员建立了深厚的个人联系。每年50名学生中有1/3是PMDA的年轻审评员，此外，培训课程的20名辅导员中有1/3是PMDA经验丰富的审评员。2004年，他加入了礼来亚洲，在神户的药品开发部门日本法规事务团队中担任法规科学家，九年后晋升为高级法规科学家。2019年，他加入Amicus Theconsultingpeutics，在东京担任法规事务高级经理，并于2022年晋升为法规事务副总监。2024年6月，东内祥浩先生开始了自雇生涯。高级日语译者简介文英淑女士近年来，泰格医药大力开拓日本医药市场，已成功助力多家中国生物制药公司的新药在日本成功上市，同时也积极引入日本企业到中国发展。文英淑目前担任泰格医药日本公司副总裁，负责日本制药/生物科技公司的临床试验业务开发及泰格医药集团在日本的业务发展，是一位具备战略规划、关系建立、出色沟通和批判性思维能力的专业人士，并精通中文、英文、日语和韩语。她熟练掌握领导力，专注于团队协作和流程管理。此前，她在春天医药担任日本办负责人。在加入春天医药之前，文英淑在多家大型知名日企如NEC、SONY担任高管。致谢我们衷心感谢中国医药创新促进会、上海市生物医药科技产业促进中心、泰格医药作为本栏目联合主办单位，并给予我们宝贵的建议和支持。往期回顾第1期(系列A-1):医药研发达人中文版“出海日本”首期:日本临床试验的历史(上)第2期(系列B-1):日本药监部门的全面介绍｜医药研发达人中文版“出海日本”第2期第3期(系列A-2):ICH E17实施后两级分化的东亚临床试验格局｜日本临床试验的历史(下)第4期(系列C-1):宇山佳明:PMDA监管科学的灵魂人物第5期(系列B-2):聚焦日本药品监管法规:药事法、药械法第6期(系列A-3):日本独有的临床试验信息登记网站挑战第7期(系列B-3):终于出来了！PMDA咨询深度指南第8期(系列A-4):日本的J-GCP与ICH-GCP有什么区别？ICF有哪些日本特色？第9期(系列C-2):中村健一:日本国立癌症研究中心如何改变亚洲临床试验格局第10期(系列B-4):PMDA如何鼓励罕见疾病药物的开发？第11期(系列A-5):日本临床试验IRB:通往单一IRB、国际化与电子化的期待之路编辑 | 毛冬蕾mao.donglei@PharmaDJ.com编辑 | 高野哲臣t2.takano@outlook.com总第2485期访问研发客网站，深度报道每日新闻抢鲜看www.PharmaDJ.com

临床1期申请上市临床结果临床申请

2025-05-01

·PRNewswire

EPIDIOLEX Continues to Lead the Way in Cannabinoid-Based Epilepsy Treatment | DelveInsight

EPIDIOLEX has significant market potential due to the growing demand for alternative therapies and its effectiveness in treating rare seizure disorders like Lennox-Gastaut syndrome and Dravet syndrome. As awareness around the benefits of CBD in treating neurological conditions increases, EPIDIOLEX stands to benefit from expanding indications and market reach. LAS VEGAS, May 1, 2025 /PRNewswire/ -- DelveInsight's ' EPIDIOLEX Market Size, Forecast, and Market Insight Report' highlights the details around EPIDIOLEX, the first FDA-approved prescription cannabidiol (CBD) to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 1 year of age or older. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EPIDIOLEX. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Jazz Pharmaceuticals' EPIDIOLEX (cannabidiol) Overview EPIDIOLEX (formerly known as GWP42003-P and marketed as EPIDYOLEX in Europe) is the first plant-based, prescription cannabis-derived oral medication developed by GW Pharmaceuticals. It represents a new category of antiepileptic drugs with a unique mechanism of action. The active compound, cannabidiol (CBD), is a naturally occurring cannabinoid found in the Cannabis sativa L. plant. While the exact way EPIDIOLEX works to reduce seizures in humans remains unclear, it is distinct from traditional anticonvulsants. CBD does not act through CB1 receptors or by blocking voltage-gated sodium channels, which are typical pathways for other antiepileptic drugs. Instead, CBD is thought to work by influencing multiple internal pathways, potentially producing a combined anticonvulsant effect. These include enhancing neuronal inhibition (through synaptic and extrasynaptic GABA channels), affecting intracellular calcium levels (via TRPV channels, VDAC, and GPR55), and reducing inflammation, possibly via adenosine modulation. Importantly, CBD does not directly bind to or activate the CB1 or CB2 receptors at levels relevant to its seizure-reducing effects. Ongoing research is examining other possible mechanisms such as interactions with adenosine, glycine, GABAergic systems, and serotonin receptors. EPIDIOLEX has received regulatory approval in numerous countries, including the U.S. (FDA), Europe (European Commission), Great Britain (MHRA), Australia (Therapeutic Goods Administration), Switzerland (Swissmedic), Israel (Ministry of Health), and New Zealand (Medicines and Medical Devices Safety Authority). It is formulated as an oral solution containing highly purified CBD. In the U.S., it is approved to treat seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients aged one year and older. In the European Union, under the name Epidyolex, it is approved as an add-on treatment with clobazam for LGS and DS in patients two years and older, and for TSC-associated seizures as well. Both the FDA and EMA have granted Orphan Drug Designation (ODD) for EPIDIOLEX/EPIDYOLEX for these conditions. EPIDIOLEX/EPIDYOLEX net product sales rose by 15% to USD 972.4 million in 2024 and by 14% to USD 275.0 million in the fourth quarter of 2024, compared to the corresponding periods in 2023. Learn more about EPIDIOLEX/EPIDYOLEX projected market size for DEE @ EPIDIOLEX Market Potential Developmental and epileptic encephalopathies (DEE) encompass a group of severe neurological conditions marked by developmental delays and persistent epilepsy. These disorders typically present early in life and are linked to significant cognitive deficits. According to DelveInsight's analysis, there were approximately 287K diagnosed prevalent cases of DEE across the 7MM in 2024. This number is projected to increase due to the rise in neurological disorders, including epilepsy, as well as environmental and maternal health factors contributing to a higher DEE prevalence throughout the forecast period (2025−2034). The US FDA has approved a range of medications for conditions like LGS, Dravet Syndrome, and CDKL5 Deficiency Disorder, including various drug classes such as sodium channel modulators, GABA receptor modulators, calcium channel blockers, receptor blockers, and others. Some of the available anti-epileptic drugs (AEDs) include FINTEPLA, EPIDIOLEX, TOPAMAX, BANZEL, LAMICTAL, FELBATOL, ONFI, KLONOPIN, DIACOMIT, SABRIL, and more. In 2024, the total market size for DEE in the 7MM was around USD 2.3 billion, and this figure is expected to grow by 2034, driven by wider adoption of approved therapies due to label expansions and the introduction of emerging treatments. Discover more about the DEE market in detail @ Developmental and Epileptic Encephalopathy Market Report Emerging Competitors of EPIDIOLEX Some of the drugs in the pipeline include EPX-100 (Harmony Biosciences), Bexicaserin (Lundbeck), Zorevunersen (Stoke Therapeutics/Biogen), Relutrigine (Praxis Precision Medicines), and others. In August 2024, the FDA removed a partial clinical hold as the company advances toward a Phase III registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome. In July 2024, Longboard Pharmaceuticals announced that the FDA had granted Breakthrough Therapy designation for its investigational drug bexicaserin for the treatment of seizures associated with DEEs for patients 2 years of age or older. To know more about the number of competing drugs in development, visit @ EPIDIOLEX Alternatives Key Milestones of EPIDIOLEX In August 2024, Jazz Pharmaceuticals reported top-line results from a Phase 3 open-label, single-arm study conducted in Japan, assessing the safety and effectiveness of its cannabidiol oral solution (marketed globally as EPIDIOLEX/EPIDYOLEX) as an add-on therapy for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). While the trial did not achieve its primary efficacy goal—defined as a pre-specified percentage reduction in seizure frequency during the up-to-16-week treatment period compared to baseline in Japanese pediatric patients—numerical improvements were noted in both the primary and several secondary measures. The study did not identify any new safety concerns. In May 2021, Jazz Pharmaceuticals acquired EPIDIOLEX as part of the acquisition of GW Pharmaceuticals plc, or GW, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. In April 2021, EPIDYOLEX was approved for adjunctive therapy of seizures associated with TSC for patients 2 years of age and older in the EU. In July 2020, EPIDYOLEX was approved for the treatment of seizures associated with TSC in patients one year of age and older. FDA also approved the expansion of all existing indications, LGS and DS, to patients one year of age and older. In September 2019, the European Commission approved EPIDYOLEX for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. In June 2018, EPIDIOLEX was approved in the US for the treatment of seizures associated with two rare and severe forms of epilepsy, LGS and DS, in patients two years of age and older. EPIDIOLEX Launch Status EPIDIOLEX was launched on November 1, 2018, in the US market after FDA approval for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older. EPIDYOLEX was launched in 2019 in Germany and the UK market for use as adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older (GW Pharmaceuticals, 2019b). In 2021, EPIDYOLEX was launched in Spain. In the first quarter of 2022, the company launched EPIDYOLEX for LGS, Dravet syndrome, and TSC in Ireland, for TSC in Scotland and Wales. In the third quarter of 2022, the company launched EPIDYOLEX for TSC in Italy and Switzerland. In the fourth quarter of 2022, the company successfully completed the pricing and reimbursement process and commercial launch of EPIDYOLEX in France. Discover how EPIDIOLEX/EPIDYOLEX is shaping the DEE treatment landscape @ EPIDIOLEX CBD EPIDIOLEX Market Dynamics EPIDIOLEX, the first FDA-approved cannabidiol (CBD)-based medication for the treatment of rare forms of epilepsy, has had a significant impact on the pharmaceutical market since its approval in 2018. The drug is primarily used to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy. The market dynamic for EPIDIOLEX is shaped by several factors, including increasing awareness of its therapeutic potential, the expanding medical cannabis industry, and the growing demand for alternative treatments to traditional anti-epileptic drugs. One of the main drivers of the EPIDIOLEX market is the rising acceptance of CBD-based therapies. As medical cannabis becomes more mainstream and research into its medicinal properties continues to grow, patients and healthcare providers are increasingly turning to EPIDIOLEX as a treatment option. The growing body of clinical evidence supporting its effectiveness and safety has reinforced this trend. Additionally, as the stigma around cannabis-derived medications continues to fade, it opens up more opportunities for EPIDIOLEX in both pediatric and adult patient populations. However, the market for EPIDIOLEX is not without its challenges. High treatment costs and insurance coverage issues can limit accessibility for some patients, especially in markets outside the United States. Despite its clinical efficacy, the price point for EPIDIOLEX remains a point of contention, as it may not be affordable for all families, especially when compared to other conventional anti-epileptic drugs. Furthermore, competition from other cannabinoid-based therapies, as well as new developments in the broader epilepsy drug market, could impact the drug's market share over time. Looking ahead, the market dynamics for EPIDIOLEX will likely continue to evolve as the global regulatory landscape changes and new clinical trials further establish its benefits in other neurological conditions. With increasing research into CBD's potential applications, there may be opportunities for EPIDIOLEX to expand its indications, providing a solid foundation for its continued growth. However, market growth will also depend on the ability to address pricing concerns, improve insurance coverage, and navigate potential competitive pressures from both traditional and emerging therapies. Dive deeper to get more insight into EPIDIOLEX/EPIDYOLEX's strengths & weaknesses relative to competitors @ EPIDIOLEX Market Drug Report Table of Contents Related Reports Developmental and Epileptic Encephalopathies Market Developmental and Epileptic Encephalopathies Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DEE companies, including vid Therapeutics, Stoke Therapeutics, Biogen, Harmony Biosciences, Lundbeck (Longboard Pharmaceuticals), Praxis Precision Medicines, SK Life Science, Cerecin Neurosciences, Encoded Therapeutics, Praxis Precision Medicines, among others. Developmental and Epileptic Encephalopathies Pipeline Developmental and Epileptic Encephalopathies Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key DEE companies, including Denovo Biopharma, Cantex Pharmaceuticals, CNS Pharmaceuticals, CANbridge Pharmaceuticals, Vaximm, Inovio Pharmaceuticals, Mustang Bio, Bullfrog AI Holdings, Cantex, Chimeric Therapeutics, Philogen, Boehringer Ingelheim, Photonamic GmbH, Berg Pharma, Beyond Bio, Genenta Science, Polaris Pharmaceuticals, Telix Pharmaceuticals, Shanghai Simnova Biotechnology, NEONC Technologies, among others. Dravet Syndrome Market Dravet Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Dravet syndrome companies including Biocodex, GW Pharmaceuticals, Zogenix, Ovid Therapeutics, PTC Therapeutics, among others. Lennox-Gastaut Syndrome Market Lennox-Gastaut Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Lennox-Gastaut syndrome companies, including GlaxoSmithKline, Meda Pharmaceuticals, Roche, Lundbeck, Greenwich Biosciences, Janssen Pharmaceuticals, Eisai, Zogenix, Takeda, Ovid Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准临床3期并购突破性疗法孤儿药

2025-04-24

·研发客

终于出来了！PMDA咨询深度指南

撰文｜东内祥浩（WaRA Consulting）翻译｜文英淑（泰格医药日本公司副总裁）中文版翻译负责人｜医药研发达人主编　高野哲臣（t2T Healthcare股份公司总裁兼首席执行官）（2025年4月25日出版，医药研发达人中文版“出海日本”第7期，系列B《日本的监管制度及其实际情况》第三篇） // •虽然PMDA咨询（相当于中国国家药监局药品审评中心开展的沟通交流）并非强制要求，然而，PMDA的创立目的是为了提供从临床试验启动前到审批为止的整体指导和审评，确保药品、医疗器械等的质量、有效性和安全性。因此，接受PMDA的建议对于减少审评期间的问询和开发品种尽早获得批准非常有帮助。•PMDA咨询中所提供的建议均是从注册申请品种的审评角度给出的。这意味着，咨询申请人按照PMDA的建议进行研发，将更有可能符合PMDA的审评标准并获得批准。•在所有阶段开展PMDA咨询需要投入大量的时间和金钱，因此咨询申请人需要根据研究药物的情况充分考虑何时进行PMDA咨询。根据笔者经验，至少在开展日本人受试者的临床试验启动前（如Ⅰ期临床试验启动前或Ⅱ期临床试验启动前）以及Ⅱ期临床试验结束后进行咨询，以得到PMDA关于开发方针的认同，是一个较为合理的选择。本连载的系列B将介绍日本药品监管制度的基本信息。这一期将谈一谈PMDA咨询。1. PMDA咨询的特点PMDA为开发药品等产品的公司、大学和研究机构提供有关开发方针、临床试验内容、申报资料等方面的指导和建议。因为与PMDA主要就临床试验相关的事宜进行面对面的咨询，从而获得相应的建议，因此这被称为“面对面建议”或“临床试验咨询”。在本文中，笔者将称之为“PMDA咨询”。其特点如下：① 结合咨询申请人的需求，根据开发阶段，PMDA细致的应对各种类型的咨询内容。② 在提供新药临床试验咨询时，PMDA将考虑拟进行临床试验的伦理性、科学性、可靠性及受试者的安全性，确认其是否符合注册申请的要求，并提供旨在提高临床试验质量的建议等。③ PMDA为大学、研究机构等学术机构以及新兴药企、风险投资企业提供临床开发初期所需的临床试验计划制定等相关的指导和建议。这被称为监管科学（Regulatory Science, 以下简称RS）战略咨询。④ 此外，PMDA还提供“简单咨询”，用于确认注册申请的申请类别等简单问题，以及“咨询前面谈”等，用于提前整理咨询内容等，以促进 PMDA 咨询顺利进行。笔者建议在申请任何PMDA咨询之前进行咨询前面谈。在咨询前面谈中，不仅可以整理咨询中需要讨论的问题，还可以捕捉到PMDA对所咨询事项的接受难易度以及解决问题的关键点。能否成功捕捉PMDA的想法，取决于制药企业注册负责人员的经验技能（参见笔者精选信息4）。笔者精选信息4咨询前面谈的重要性在咨询前面谈时，PMDA将对咨询申请人拟咨询的事项表达其意见。例如，当咨询事项为确认临床试验方案中设定的主要终点的妥当性时，PMDA会在咨询前面谈中要求说明设定主要终点的根据，包括其与目标疾病的相关性以及它将带来的临床上的改善意义。在这种情况下，PMDA要求的信息需要体现在后续的正式PMDA咨询（正式咨询）时的咨询资料中。更重要的是，如果PMDA认为咨询申请人拟设定的主要终点经不起审评过程中针对科学性的推敲，则会在咨询前面谈时提出非常严苛的要求。如果咨询申请人预判在正式咨询资料中给不出满足严苛要求的内容时，变更主要终点可能是明智之举。如果在与 PMDA的咨询前面谈中无法捕捉到PMDA对变更与否的看法，而维持原有的主要终点直接进入正式PMDA咨询时会面临被拒绝的风险。在PMDA咨询期间，万一PMDA给出的官方结论表明无法接受主要终点时，咨询申请人需要更改主要终点后再次申请PMDA咨询，以确认修改后的主要终点的可接受度，开发周期也随之延长。2. PMDA咨询的类型和实施时间如上所述，根据咨询的目的和内容，PMDA咨询有详细的分类，本文将重点介绍对药物研发至关重要的部分。图1显示了与药物研发相关的PMDA咨询的主要类型及其实施时间。图1. 药物研发相关的PMDA咨询的主要类型与实施时间（1）临床试验相关的咨询① Ⅰ期临床试验启动前咨询根据前期获得的质量、安全性、药理学、药代动力学、外国人体使用经验、国外批准情况、同类药品等信息，提供I期临床试验的研究设计等方面的指导和建议。② Ⅱ期临床试验启动前咨询在Ⅰ期临床试验结束到确定临床推荐剂量前，根据已获得的Ⅰ期临床试验等的数据、外国人体使用经验、国外批准情况、同类药品等信息，提供Ⅱ期临床试验的研究设计（剂量探索试验）等方面的指导和建议。③ Ⅱ期临床试验结束后咨询确定临床推荐剂量后，根据已实施的临床试验结果和类似药物的信息，就试验用药品的Ⅲ期临床试验的研究设计提供指导和建议。除了咨询Ⅲ期临床试验的研究设计外，此次咨询还需要确认注册申请时需提交的临床数据集（完整的临床数据）以及注册所需的电子数据集。也就是说，除了Ⅲ期临床试验的结果符合预期以外，还需要哪些试验才可以满足注册申请的要求？在此次咨询确认获批所需其他试验非常关键。如果忽视这一点，当PMDA在注册申请前咨询或提交注册申请后指出缺少数据时，将会浪费大量时间。④ 注册申请前咨询当临床开发已经完成或即将完成，正在准备注册申请所需的申报资料时，PMDA将结合迄今为止的临床试验结果，就如何撰写申报资料以及资料是否充分提供指导和建议。根据笔者的经验，如果是按照Ⅱ期临床试验结束后咨询的内容开展的Ⅲ期试验，且结果符合预期，通常会跳过注册申请前咨询，直接递交注册申请。另一方面，即使Ⅲ期临床试验的结果不如预期好，但如果能够解释该研究药物的临床意义，并且申请人认为它是患者必需的产品，则可以在注册申请前咨询中询问PMDA是否批准。（2）监管科学（RS）战略咨询PMDA主要向大学、研究机构和新兴药企、风险投资企业，提供从药品、医疗器械、再生医疗等产品的开发产品候选物确定的最终阶段到临床开发初期【直至概念验证试验（相当于Ⅱ期早期试验）】所需的临床试验计划制定等相关的指导和建议。① RS 综合咨询PMDA主要面向不熟悉咨询及注册申请的人员，确认拟咨询的内容是否适合进行RS战略咨询，并说明RS战略咨询业务的内容和程序。针对药械法的制度，以及PMDA开展的审评审批等相关问题给予一般性的说明。① RS战略咨询PMDA相关审评部门的审评小组审查咨询申请人提交的材料后，告知PMDA对拟开展的临床试验及注册申请的各类咨询事项的官方意见以及所提供的具体的指导和建议。3. 从申请PMDA咨询到接收咨询记录的流程图2显示了从申请PMDA咨询到接收咨询记录的流程。图2. 从申请PMDA咨询到接收咨询记录的流程（1）咨询前面谈申请在希望咨询前面谈的大约两周前，咨询申请人需要向PMDA提交咨询前面谈申请书，在申请书中填写目标疾病、所开发品种的信息，以及希望在PMDA咨询时确认的事项。需要注意的是，即使描述或附加已经获得的数据，PMDA也不会在咨询前面谈时评估这些数据，所以咨询申请人不会得到PMDA对这些数据的反馈意见。提交申请书几天后，PMDA负责部门的联络人会通知咨询申请人几个备选日程，咨询申请人协调日程后，确定咨询前面谈的时间。咨询前面谈通常在PMDA内的小型会议室进行。面谈时间大约为20~30分钟，参加咨询前面谈的人数一般限制为5人。但如果希望更多人参加，可与PMDA负责部门的联络人协商。咨询前面谈不收取任何费用。根据笔者以往的经验，即便在咨询前面谈中大致了解了PMDA的想法，也不建议跳过后续的正式PMDA咨询（正式咨询）。原因是咨询前面谈不会进行数据评估，而只是针对PMDA咨询时需要讨论的事项给出咨询资料应包含哪些内容的一般性建议。为了获取该研究药物审评审批相关的PMDA的官方意见，PMDA咨询是必不可少的。（2）从咨询前面谈到申请PMDA咨询日程的协调如“笔者精选信息4”中所述，咨询申请人将根据咨询前面谈的结果决定正式PMDA咨询的日期。原则上，需要在希望进行PMDA咨询月份的两个月前的第一个工作日（上午10点到下午4点）提交PMDA咨询日程的协调申请。申请后5个工作日内会收到来自PMDA负责部门的联络人针对咨询日期的答复。之后，咨询申请人需要在15个工作日内支付咨询费用并提交PMDA咨询申请书。咨询费用的收费标准从Ⅰ期临床试验启动前的约460万日元（根据近期汇率，大致等于23.6万元人民币）到注册申请前的约950万日元（根据近期汇率，大致等于45.6万元人民币）不等。具体金额，请参阅PMDA网站（日语）。 https://www.pmda.go.jp/files/000272392.pdf（3）从提交咨询资料到实施PMDA咨询咨询申请人需根据咨询前面谈中得到的建议准备咨询资料，并在PMDA咨询日期的约5周前的星期一提交咨询资料约两周后，PMDA将发出针对咨询资料内容的问询事项，而咨询申请人需在一周内答复相关事项。在答复1~2周后，PMDA将以电子邮件的形式发送针对咨询事项的意见（PMDA的初步意见）。咨询申请人需要在1周内对该初步意见作出答复，或给出同意初步意见的反馈，或给出不同意初步意见并对PMDA未理解之处进行补充说明的反馈。PMDA咨询当天面对面咨询的流程如下：① 与会人员互相介绍② 咨询申请人陈述（对PMDA的初步意见的反馈）及问答环节③ 讨论咨询的事项④ 总结如果咨询申请人在PMDA咨询前收到的初步意见中了解了PMDA对所有咨询事项的立场并且没有异议，咨询申请人可以取消面对面的PMDA咨询，仅接收咨询记录。相反，只要咨询申请人不取消PMDA咨询，将如期举行面对面的咨询（包括线上会议）。也就是说，在日本，不会出现违背咨询申请人的意愿而无法进行咨询申请人与监管机构之间的直接对话的情况。此外，如果在PMDA咨询时，有无法当场给出结论的问题，则双方可以协商将问题带回各自讨论后，再次安排与PMDA的咨询（事后咨询）。（4）撰写咨询记录PMDA咨询结束后2~3周，PMDA将发送一份咨询记录草案。咨询申请人需要仔细阅读咨询记录草案，对任何含糊不清或与咨询申请人见解不同的陈述，都要寻求PMDA的解释或要求修改相关内容。咨询记录将在PMDA咨询后30个工作日内定稿。咨询记录的开头将记载如下固定语句：“请注意，本咨询记录中所示的判断和其他决定是基于所提交的资料和实施PMDA咨询时的科学水平做出的。因此，本咨询记录中所示的判断和其他决定的妥当性可能会根据新获得的知识和科学进步等而发生变化。”其字面意思可以理解为，通过PMDA咨询获得的建议并不能保证100%获得批准。以笔者的经验来看，世上没有100%的事情，所以PMDA特意加了这段声明。因为PMDA对PMDA咨询时的所表达的意见负有重大责任，一般不会出现违背咨询建议而进行审评的情形。另一方面，咨询申请人在PMDA咨询时的陈述也将被谨慎对待，当递交的注册申请内容与PMDA咨询记录不一致时，则很可能会被视为审评中重点讨论的事项，审评工作也极有可能无法继续进行。因此，在PMDA咨询后需要对计划作出重大变更时，建议就该点进行“追加咨询”并得到PMDA的认可。4. 总结由于PMDA咨询不是强制性的，因此咨询申请人可以跳过研发阶段的咨询而直接递交注册申请。然而，PMDA创立的目的是针对药品、医疗器械等的质量、有效性和安全性，提供从临床试验启动前到审批为止一脉相承的指导和审评。因此，PMDA咨询中所提供的建议，均是从注册申请品种的审评角度给出的，咨询申请人按照PMDA的建议进行研发将减少PMDA在审评期间的问询，对于尽早获得批准非常有帮助。在所有阶段开展PMDA咨询需要投入大量的时间和金钱，因此咨询申请人需要根据研究药物的情况充分考虑何时进行PMDA咨询。根据笔者的经验，至少在开展日本人受试者的临床试验启动前（Ⅰ期临床试验启动前或Ⅱ期临床试验启动前）以及Ⅱ期临床试验结束后进行咨询，得到PMDA关于开发方针的认同。预告计划2025年5月上旬出版的第8期将是由高野哲臣先生撰写的系列A的第四篇，ICH-GCP 与 J-GCP 之间的差异、日本独有的共通的ICF模板。中文版第7期作者简介东内祥浩先生（Yoshihiro Higashiuchi）东内祥浩先生是一位拥有20年经验的资深监管事务顾问、监管科学家，负责领导监管活动，包括与药品医疗器械综合机构（PMDA）的咨询、新药申请（NDA）提交和新分子实体（NME）、新适应症/产品线扩展（NILEX）的监管批准，其中包括5个NME和3个NILEX的监管批准。通过13年（自2008年起）组织策划日本药物信息协会（DIA）监管事务培训课程的活动，东内祥浩与PMDA的审评员建立了深厚的个人联系。每年50名学生中有1/3是PMDA的年轻审评员，此外，培训课程的20名辅导员中有1/3是PMDA经验丰富的审评员。2004年，他加入了礼来亚洲，在神户的药品开发部门日本法规事务团队中担任法规科学家，九年后晋升为高级法规科学家。2019年，他加入Amicus Theconsultingpeutics，在东京担任法规事务高级经理，并于2022年晋升为法规事务副总监。2024年6月，东内祥浩先生开始了自雇生涯。高级日语译者简介文英淑女士近年来，泰格医药大力开拓日本医药市场，已成功助力多家中国生物制药公司的新药在日本成功上市，同时也积极引入日本企业到中国发展。文英淑目前担任泰格医药日本公司副总裁，负责日本制药/生物科技公司的临床试验业务开发及泰格医药集团在日本的业务发展，是一位具备战略规划、关系建立、出色沟通和批判性思维能力的专业人士，并精通中文、英文、日语和韩语。她熟练掌握领导力，专注于团队协作和流程管理。此前，她在春天医药担任日本办负责人。在加入春天医药之前，文英淑在多家大型知名日企如NEC、SONY担任高管。致谢我们衷心感谢中国医药创新促进会、上海市生物医药科技产业促进中心、泰格医药作为本栏目联合主办单位，并给予我们宝贵的建议和支持。编辑 | 毛冬蕾mao.donglei@PharmaDJ.com编辑 | 高野哲臣t2.takano@outlook.com总第2410期访问研发客网站，深度报道每日新闻抢鲜看www.PharmaDJ.com

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