移植治疗联盟收到FDA关于iBox评分系统全面资格认证包的可审评通知-动脉网

Transplant Therapeutics Consortium Receives FDA Notice of Reviewability for the iBox Scoring System Full Qualification Package

CISION 等信源发布 2025-07-30 22:06

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The iBox Scoring System is a novel composite biomarker endpoint predictive of long-term graft survival after kidney transplantation

iBox评分系统是一种新型的复合生物标志物终点，可预测肾移植后的长期移植物存活情况。

On track to be the first qualified reasonably likely surrogate endpoint in transplant for use in the FDA accelerated approval pathway

有望成为移植领域首个合格的、合理的、可能的替代终点，用于FDA加速批准途径。

FDA aims for a 10 month review of the Full Qualification Package

FDA的目标是对完整资格认证包进行为期10个月的审查

MOUNT LAUREL, N.J.

劳雷尔山，新泽西州

，

July 30, 2025

2025年7月30日

/PRNewswire/ -- The American Society of Transplant Surgeons (ASTS) and American Society of Transplantation (AST) announced today that the Transplant Therapeutics Consortium (TTC) received notification from FDA of the Reviewability of the Full Qualification Package (FQP) for the iBox Scoring System, a novel reasonably likely surrogate efficacy endpoint for kidney transplant clinical trials.

/PRNewswire/ -- 美国移植外科医生协会 (ASTS) 和美国移植协会 (AST) 今天宣布，移植治疗联盟 (TTC) 收到了 FDA 关于 iBox 评分系统完整资格包 (FQP) 可审性的通知，该系统是一项用于肾移植临床试验的新型很可能有效的替代终点。

The FDA Drug Development Tool qualification program aims to communicate the final decision within 10 months targeting .

FDA药物开发工具资格认证计划旨在针对目标，在10个月内传达最终决定。

April 2026

2026年4月

. The Full Qualification Package is the final step in the FDA qualification process and iBox scoring system is the first transplant endpoint to reach this stage of qualification.

完整资格认证包是FDA资格认证过程的最后一步，iBox评分系统是第一个达到此资格认证阶段的移植终点。

Improving the long-term survival of transplanted kidneys is an important area of unmet need for transplantation recipients. The current efficacy failure endpoint has typically shown non-inferiority of immunosuppressive regimens but the iBox Scoring System is proposed to be used to demonstrate the superiority of a new agents compared to the standard of care as a co-primary endpoint in pivotal drug therapeutic studies.

改善移植肾脏的长期存活率是移植受者未满足需求的重要领域。目前疗效失败终点通常显示免疫抑制方案的非劣效性，但提议使用iBox评分系统作为关键药物治疗研究的共同主要终点，以证明新药剂相对于标准治疗的优越性。

As a reasonably likely surrogate endpoint, the iBox scoring system would be used in the Accelerated Approval Pathway at the FDA designed to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need. .

作为一个相对可能的替代终点，iBox评分系统将用于FDA的加速审批通道，该通道旨在允许更早地批准治疗严重疾病并满足未满足医疗需求的药物。

'It's not surprising that using a clinical trials endpoint that is nearly four decades old has been a significant impediment to the development of new therapeutics for maintenance immunosuppression and has contributed to the relatively stagnate long-term outcomes of kidney transplants. I applaud the FDA for working with the TTC to develop the iBox as a coprimary endpoint for clinical trials in kidney transplantation.

“使用一个将近四十年历史的临床试验终点，成为开发用于维持免疫抑制的新疗法的重大障碍，并导致肾移植长期疗效相对停滞，这并不令人惊讶。我赞扬FDA与TTC合作，将iBox作为肾移植临床试验的共同主要终点进行开发。”

We have recognized the strong push from the patient community for new agents that promote both longer survival of transplanted organs and improved safety and tolerability. I hope that this is the first of many collaborative efforts to validate and implement new drug development tools to achieve that end,' .

我们已经认识到患者群体对于新药的强烈需求，这些新药既能延长移植器官的存活时间，又能提高安全性和耐受性。我希望这是众多合作努力中的第一个，以验证和实施新的药物开发工具来实现这一目标。

said

说

Kenneth Newell

肯尼斯·纽厄尔

, MD, PhD, Professor of Surgery,

医学博士，哲学博士，外科学教授，

Emory University

埃默里大学

and Executive Committee Member of the TTC.

TTC执行委员会成员。

Paul T. Conway

保罗·T·康威

, Vice President and Chair of Policy and Global Affairs, American Association of Kidney Patients (AAKP) and a 28-year kidney transplant recipient stated, 'For decades, bipartisan national policy priorities, across multiple presidential administrations and Congresses, have included transplantation and sustainable long-term transplant outcomes as a means for saving more lives, protecting livelihoods, and reducing patient and taxpayer burdens.

美国肾脏患者协会（AAKP）政策与全球事务副总裁兼主席，同时也是一名拥有28年肾移植经历的接受者表示：“几十年来，跨多届总统政府和国会的两党国家政策重点都包括移植以及可持续的长期移植效果，以此作为拯救更多生命、保护生计并减轻患者和纳税人负担的手段。”

Yet, due to policy barriers, the transplant drugs patients currently depend upon to avoid organ rejection and a return to the transplant waiting list have failed to evolve and pose serious risks to both organ recipients and grafts. AAKP is optimistic that recent FDA action on the iBox is a signal the agency is putting transplant patients first, accelerating transplant drug development and re-aligning internal FDA decision-making to better advance long-standing American policy priorities.' Conway previously served as the inaugural Chair of the FDA's Patient Engagement Advisory Committee for medical devices..

然而，由于政策障碍，患者目前依赖的防止器官排斥并避免重返移植等待名单的移植药物未能发展，并对器官接受者和移植物都构成严重风险。AAKP乐观地认为，FDA最近针对iBox采取的行动表明该机构将移植患者放在首位，加快移植药物开发，并重新调整FDA内部决策以更好地推进美国长期以来的政策重点。康韦此前曾担任FDA医疗器械患者参与咨询委员会的首届主席。

'We celebrate this step toward a regulatory process that will allow for many more drugs to enter into the transplant drug development path. The patient community has been starved of new approaches to keep their precious grafts alive and thriving for a longer time, with fewer side effects and complications.

“我们庆祝这一迈向监管进程的步骤，这将允许更多药物进入移植药物开发路径。患者群体长期以来缺乏新的方法来保持他们宝贵的移植物更长时间地存活和茁壮成长，同时减少副作用和并发症。

As a person living with transplants derived from selfless individuals, I can speak for the transplant patient community in applauding this progress toward our goal of leveraging modern day science to achieve one transplant for life,'.

作为一名接受过无私人士捐献移植器官的人，我可以代表移植患者群体对这一进展表示赞赏，我们的目标是利用现代科学实现一次移植终身受用。

said Dr.

博士说。

Karin Hehenberger

卡琳·赫嫩贝格

, President Lyfebulb, and a kidney and pancreas transplant recipient.

，Lyfebulb总裁，也是一位肾脏和胰腺移植接受者。

About iBox

关于iBox

The iBox scoring system is a composite endpoint that utilizes multiple clinically relevant features (estimated glomerular filtration rate (eGFR), proteinuria, anti-human leukocyte antigen donor-specific antibody, and kidney graft biopsy histopathology) at one-year post-transplant to predict 5-year graft survival.

iBox评分系统是一个复合终点，利用移植后一年的多个临床相关特征（估计肾小球滤过率(eGFR)、蛋白尿、抗人类白细胞抗原供体特异性抗体和肾移植活检组织病理学）来预测5年移植物存活率。

The iBox scoring system (.

iBox 评分系统 (。

NCT03474003

), has been developed and validated in a landmark publication released in the BMJ by a team of researchers led by Professor

），已在BMJ发布的一篇具有里程碑意义的出版物中得到开发和验证，该研究由教授领导的一组研究人员完成。

Alexandre Loupy

亚历山大·卢皮

in Paris French NIH (Inserm), APHP and Université Paris Cité, comprising 7557 patients from 10 academic centers from

在巴黎的法国国立卫生研究院（Inserm）、巴黎公立医院集团（APHP）和巴黎西岱大学，涵盖来自10个学术中心的7557名患者，

Europe

欧洲

and the US and 3 Randomized controlled trials.

美国和三项随机对照试验。

About FDA Accelerated Approval

关于FDA加速批准

The FDA Accelerated Approval Program is designed to facilitate and expedite development of drugs that treat serious conditions, and fill an unmet medical need based on a reasonable likely surrogate endpoint (RLSE) that is supported by strong mechanistic and/or epidemiologic rationale. The Qualification of the iBox would allow it to be used as a RLSE for accelerated approval of novel new immunosuppressants for kidney transplantation.

FDA加速审批计划旨在促进和加快治疗严重疾病药物的开发，并基于一个有强有力机制和/或流行病学理论支持的合理可能替代终点（RLSE）来满足未被满足的医疗需求。iBox的认证将允许其作为RLSE用于加速批准新型肾移植免疫抑制剂。

The Accelerated Approval program has historically been successful advancing new innovative therapies for HIV and cancer and most recently promoted the introduction of 4 new drugs for IgA nephropathy, a chronic kidney disease with no approved therapies 5 years ago before the use of proteinuria as a surrogate endpoint. .

加速批准计划在历史上成功推动了针对 HIV 和癌症的新型创新疗法，最近还促进了针对 IgA 肾病（一种慢性肾病）的四种新药的问世，在五年前还没有获批的疗法，当时使用蛋白尿作为替代终点。

About TTC

关于TTC

The

Transplant Therapeutics Consortium (TTC)

移植治疗联盟 (TTC)

is a public-private partnership with the FDA founded in 2017 by the

是一家由FDA于2017年创立的公私合作伙伴关系

American Society of Transplantation (AST)

美国移植学会 (AST)

and the

和

American Society of Transplant Surgeons (ASTS).

美国移植外科医生协会 (ASTS)。

This collaboration unites the transplant community, industry leaders, and regulatory agencies dedicated to the development of innovative tools, methods and processes for facilitating advancement of new innovative therapies to improve the lives of solid organ transplant recipients.

这一合作联合了移植界、行业领袖和监管机构，致力于开发创新的工具、方法和流程，以促进新疗法的进步，改善实体器官移植受者的生活。

This submission of the FQP was accomplished by employing the resources of the TTC members and engaging with FDA throughout the biomarker qualification process. TTC is supported through funding and input from the AST*, ASTS*, argenx*, Arkana Laboratories*, Bristol Myers Squibb*, CareDx*, Critical Path Institute, CSL, CTI Clinical Trial Services, Inc.*, Eledon*, European Society of Organ Transplantation, the U.S.

通过利用TTC成员的资源并在整个生物标志物认证过程中与FDA合作，完成了FQP的提交。TTC得到了AST*、ASTS*、argenx*、Arkana Laboratories*、Bristol Myers Squibb*、CareDx*、Critical Path Institute、CSL、CTI Clinical Trial Services, Inc.*、Eledon*、欧洲器官移植学会、美国等机构的资金和支持。

Food and Drug Administration (FDA), Hansa Biopharma*, HUS .

美国食品药品监督管理局 (FDA)，汉莎生物制药*，HUS。

Helsinki University

赫尔辛基大学

Hospital, Immucor, KU Leunven, Memo Therapeutics AG*, Natera, National Institutes of Health, Novartis, Paris Transplant Group, Pirche, Sanofi*, Takeda*, Talaris Therapeutics, Thermo Fisher Scientific, The Transplantation Society, Transplant Genomics*,

医院，Immucor，KU Leuven，Memo Therapeutics AG*，Natera，国立卫生研究院，诺华，巴黎移植集团，Pirche，赛诺菲*，武田*，Talaris Therapeutics，赛默飞世尔科技，移植学会，Transplant Genomics*

University of Manitoba

曼尼托巴大学

, and Veloxis Pharmaceuticals*. Any groups that would like to join in this effort or have information or data that may contribute to further advances, can contact JoAnn Gwynn at

，以及Veloxis Pharmaceuticals*。任何希望加入此项工作或拥有相关信息或数据可能有助于进一步发展的团体，可以联系JoAnn Gwynn at

jgwynn@myast.org

。

Denotes Current TTC Members

表示当前TTC成员

The

Transplant Therapeutics Consortium (TTC)

移植治疗联盟 (TTC)

was launched in April 2017 and co-founded by the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). TTC brings together pharmaceutical companies, diagnostic companies, academic and nonprofit partners working toward a common goal of moving the field forward toward drug development solutions in transplantation..

于2017年4月推出，由美国移植学会（AST）和美国移植外科医生学会（ASTS）共同创立。TTC汇集了制药公司、诊断公司、学术界和非营利合作伙伴，共同致力于推动移植领域向前发展，寻求药物开发解决方案。

American Society of Transplantation

美国移植学会

Founded in 1982, the

成立于1982年，

American Society of Transplantation

美国移植学会

(AST) is a non-profit, 501(c)3 organization dedicated to advancing the field of transplantation and improving patient care by promoting research, education, advocacy, organ donation, and service to the community through a lens of equity and inclusion. The society is the largest transplant organization in North America (consisting of nearly 5,000 professional members), representing a majority of the nation's medical professionals engaged in the field of transplantation..

（AST）是一个非营利性501(c)3组织，致力于通过促进研究、教育、倡导、器官捐赠以及通过公平和包容的视角为社区服务，推动移植领域的发展并改善患者护理。该学会是北美最大的移植组织（拥有近5000名专业会员），代表了美国大多数从事移植领域的医疗专业人员。

Learn more at myAST.org.

请访问 myAST.org 了解更多信息。

American Society of Transplant Surgeons

美国移植外科医生学会

The American Society of Transplant Surgeons (ASTS) was founded in 1974 by 127 surgeons to unite the professionals who were advancing the fledgling field of organ transplantation.

美国移植外科医生协会 (ASTS) 于 1974 年由 127 名外科医生创立，旨在团结那些推动器官移植这一新兴领域发展的专业人士。

From this small group of dedicated individuals, we have grown to represent over 2,000 transplant professionals who are dedicated to

从这个小团队的专注个体开始，我们已经发展到代表超过2000名致力于移植工作的专业人士，

saving and improving lives through the transformative power of organ donation and transplant surgery.

通过器官捐赠和移植手术的变革力量来拯救和改善生命。

Learn more about ASTS by visiting our website at ASTS.org.

通过访问我们的网站 ASTS.org 了解更多关于 ASTS 的信息。

MEDIA CONTACTS:

媒体联系人：

Shauna O'Brien

肖娜·奥布莱恩

– AST: