Transplant Therapeutics Consortium Receives FDA Acceptance of the Qualification Plan for IBox Scoring System
Transplant Therapeutics Consortium Receives FDA Acceptance of the Qualification Plan for IBox Scoring System
Improving the long-term survival of transplanted kidneys is an important area of unmet need for transplantation recipients, and clinical trials testing new immunosuppressive agents are needed to improve kidney transplant outcomes. Although the current efficacy failure endpoint has typically shown the non-inferiority of immunosuppressive regimens, the iBox Scoring System is proposed to be used to demonstrate the superiority of a new agents compared to the standard of care (SOC) as a co-primary endpoint in pivotal drug therapeutic studies. 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.The iBox Scoring System is a novel endpoint predictive of long-term survival after kidney transplantation
iBox評分系統是一種新穎的終點預測，可預測腎移植後的長期存活
MOUNT LAUREL, N.J., Nov. 20, 2024 /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 acceptance of the Qualification Plan for the iBox Scoring System, a novel reasonably likely surrogate efficacy endpoint for kidney transplant clinical trials. This takes us one step closer to having new therapies that better the lives of transplant recipients and marks the successful completion of step two in the three-step process for FDA biomarker qualification. The iBox scoring system is the first transplant endpoint to reach this stage of qualification.
新澤西州莫林特市，2024年11月20日 / PRNewswire/ - 美國移植外科醫生學會（ASTS）和美國移植學會（AST）今天宣佈，移植治療聯盟（TTC）收到FDA關於iBox評分系統資格計劃的認可通知，這種新型而有可能的委託有效終點已經成爲腎移植臨床試驗的合理替代終點。這讓我們離擁有改善移植受體生活的新療法更近了一步，也標誌着FDA生物標記資格三步流程中的第二步成功完成。iBox評分系統是第一個達到這一資格階段的移植終點。
"With the iBox Scoring System on the pathway to FDA qualification, we are closer than ever to cutting-edge clinical tools that could extend the lives of transplant recipients. iBox is opening the door to using artificial intelligence and machine learning. This achievement signals a transformative shift in how we approach clinical trials for transplant patients and is a critical step forward in modernizing the field," said AST President Jon Kobashigawa, MD.
「隨着iBox評分系統走上FDA資格之路，我們比以往任何時候都更接近尖端臨床工具，這些工具有望延長移植受體的生命。iBox正在開啓使用人工智能和機器學習的大門。這一成就標誌着我們在如何對待移植患者的臨床試驗方面發生了變革，並且是現代化該領域的關鍵步驟，」美國移植學會會長喬恩·小林川川博士說。
Improving the long-term survival of transplanted kidneys is an important area of unmet need for transplantation recipients, and clinical trials testing new immunosuppressive agents are needed to improve kidney transplant outcomes. Although the current efficacy failure endpoint has typically shown the non-inferiority of immunosuppressive regimens, the iBox Scoring System is proposed to be used to demonstrate the superiority of a new agents compared to the standard of care (SOC) as a co-primary endpoint in pivotal drug therapeutic studies. 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.
改善移植腎的長期存活是移植受體一個重要的未滿足需求領域，需要進行測試新的免疫抑制藥物的臨床試驗，以改善腎移植結果。儘管當前的有效性失敗終點通常表明免疫抑制方案的非劣效性，但建議使用iBox評分系統來證明新藥物與護理標準（SOC）相比的優越性作爲關鍵藥物治療研究中的共同主要終點。作爲合理可能的替代終點，iBox評分系統將在FDA的加速批准路徑中使用，該路徑旨在允許提前批准治療嚴重疾病並填補未滿足醫療需求的藥物。
"We are delighted that the iBox which received endorsement and qualification by the European Medicines Agency (EMA) in 2022 now also has received acceptance of the FDA Biomarker Qualification Plan as an efficacy endpoint for kidney transplant clinical trials. This acceptance will facilitate more rapid assessment of novel Immunotherapeutics for kidney transplant recipients and harmonization of multinational clinical trials. We are grateful to our many partners and collaborators in the Transplant Therapeutics Consortium for their steadfast support of this important initiative to facilitate innovation in transplantation," said ASTS President Ginny Bumgardner.
「我們非常高興，2022年歐洲藥品管理局（EMA）認可並資格認證的iBox現在也已獲得FDA生物標誌物資格計劃的接受，作爲腎移植臨床試驗的有效終點。這一接受將促進更快地評估腎移植受體的新型免疫治療藥物，並協調跨國臨床試驗。我們感謝移植治療學聯盟中許多合作伙伴和合作者對這一促進移植創新的重要舉措的堅定支持，」ASTS主席吉尼·邦加德納博士表示。
"We desperately need new regulatory pathways which bring investment into transplantation. New therapies are essential to prolong the life of transplant recipients and take the best care of the gift of life provided by our donors" said Dr. Karin Hehenberger, President Lyfebulb, and a kidney and pancreas transplant recipient. "This step is critical to enable the dream of 'One Kidney for Life' to become a reality."
「我們迫切需要引入投資進入移植領域的新監管路徑。新療法對延長移植受體的生命至關重要，並最好地照顧我們的捐贈者提供的生命禮物，」Lyfebulb主席、腎臟和胰腺移植受體卡琳·海恩貝格爾博士表示。「這一步驟對實現『一生只要一個腎』的夢想至關重要。」
The iBox scoring system is a composite endpoint used as a risk prediction tool 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. The iBox Qualification Plan is supported by strong mechanistic and epidemiologic data of the association with late kidney functional decline and failure and includes data from single center and randomized controlled trials representing over 2,500 de novo kidney transplant recipients.
iBox評分系統是一個綜合終點，作爲風險預測工具，利用多個臨床相關特徵（估算的腎小球濾過率（eGFR）、蛋白尿、抗人白細胞抗原特異性抗體和腎臟移植活檢病理學）在移植後一年預測5年移植物存活率。iBox資格計劃得到了與晚期腎功能下降和衰竭相關的強有力機制和流行病學數據的支持，幷包括來自代表2500多個新的腎臟移植受體的單箇中心和隨機對照試驗的數據。
The iBox scoring system (NCT03474003), has been developed and validated in a landmark publication released in the BMJ by a team of researcher led by Pr Alexandre Loupy in Paris French NIH (Inserm), APHP and Université Paris Cité, comprising 7557 patients from 10 academic centres from Europe and the US and 3 RCT's. iBox leveraged high quality longitudinal data to build a robust predictive system for long-term kidney allograft loss. iBox has been extensively validated for use in the treatment of individual patients in the clinical care setting and common clinical scenarios including response to treatment in in T-cell mediated rejection, antibody-mediated rejection and immunosuppressive regimen minimization (BMJ 2019; 366:l4923).
iBox評分系統（NCT03474003），由巴黎法國國立衛生研究院（Inserm）、APHP和巴黎城市大學的Alexandre Loupy教授領導的團隊在BMJ上發佈的一篇重要文章中開發和驗證，共涵蓋了來自歐洲和美國的10個學術中心和3個RCT的7557名患者。iBox利用高質量的縱向數據構建了一個強大的預測系統，用於預測長期腎移植物喪失。iBox已廣泛驗證，可用於在臨床護理環境中治療個體患者和常見的臨床病例，包括對T細胞介導的排斥、抗體介導的排斥和免疫抑制方案最小化治療反應的檢查（BMJ 2019；366：l4923）。
The Transplant Therapeutics Consortium (TTC) is a public-private partnership founded in 2017 by the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). This collaboration unites the transplant community, industry leaders, and regulatory agencies to foster consensus-driven, data-focused research aimed at enhancing the development of innovative tools and methods for evaluating the safety and efficacy of solid organ transplants (SOT).
移植治療財團（TTC）是美國移植學會（AST）和美國移植外科醫生學會（ASTS）於2017年創立的公私合作伙伴關係。這種合作將整合移植社區、行業領袖和監管機構的力量，推動以共識爲基礎、數據爲導向的研究，旨在提升固體器官移植（SOT）安全性和有效性評估方法的創新工具和方法的發展。
This regulatory qualification achievement 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. Food and Drug Administration (FDA), Hansa Biopharma*, HUS Helsinki University 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*, University of Manitoba, and Veloxis Pharmaceuticals*.
這一監管資格取得是通過利用TTC成員的資源，並在整個生物標誌物資格過程中與FDA合作而實現的。TTC得到AST*、ASTS*、argenx*、Arkana Laboratories*、Bristol Myers Squibb*、caredx*、Critical Path Institute、CSL、CTI臨床試驗服務公司*、Eledon*、歐洲器官移植學會、美國食品藥品監督管理局（FDA）、海莎生物製藥*、HUS赫爾辛基大學醫院、Immucor、KU列文大學、Memo Therapeutics AG*、natera、美國國家衛生研究所、諾華、巴黎移植小組、Pirche、賽諾菲安萬特*、武田*、Talaris Therapeutics、賽默飛世爾、移植學會、移植基因組學*、曼尼託巴大學和Veloxis Pharmaceuticals*的資金支持和意見。
TTC is working on its next regulatory milestone, the submission of the Full Qualification Package for regulatory endorsement of the iBox Scoring System as a reasonably likely surrogate endpoint by the FDA.
TTC正在努力實現下一個監管里程碑，即提交iBox評分系統的全面資格包，以獲得FDA對其作爲相對可能的替代終點的認可。
As part of the 21st Century Cures Act, passed into law in December 2016, public-private partnerships consisting of government entities, including FDA, the biopharmaceutical industry, health care providers, academic researchers, and patient advocacy organizations, have been encouraged to work together to foster innovation in the development of new therapies by qualifying new drug development tools that can accelerate the process of making new therapies available to patients. For more information, click here. 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 [email protected].
作爲2016年12月通過的《21世紀醫治法案》的一部分，政府實體、包括FDA、生物製藥行業、醫療保健提供者、學術研究人員和患者倡導組織等組成的公私合作伙伴關係得到鼓勵，共同促進新療法開發的創新，通過評定新藥開發工具，以加速使新療法面向患者。欲了解更多信息，請點擊這裏。任何希望加入此努力或具有可能促進進一步進展的信息或數據的團體，可以通過[email protected]聯繫JoAnn Gwynn。
The Transplant Therapeutics Consortium (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.
移植治療協會(TTC)於2017年4月啓動，由美國移植學會(AST)和美國移植外科醫師學會(ASTS)共同創立。TTC彙集了藥品公司、診斷公司、學術和非營利夥伴，共同致力於向前推進領域，推動移植領域的藥物研發解決方案。
American Society of Transplantation
Founded in 1982, the American Society of Transplantation (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.
美國移植學會
成立於1982年，美國移植學會(AST)是一家非營利性的501(c)3組織，致力於推動移植領域的發展，並通過促進研究、教育、倡導、器官捐獻和爲社區提供服務來改善患者護理，以平等和包容的視角。該學會是北美最大的移植組織(擁有近5000名專業會員)，代表着該領域從事工作的絕大多數全國醫療專業人員。
Learn more at myAST.org.
請在myASt.org了解更多。
American Society of Transplant Surgeons
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.
美國移植外科醫師學會
美國移植外科醫師學會(ASTS)成立於1974年，由127名外科醫師聯合成立，旨在團結那些正在推動新生器官移植領域發展的專業人員。
From this small group of dedicated individuals, we have grown to represent over 2,000 transplant professionals who are dedicated to saving and improving lives through the transformative power of organ donation and transplant surgery. Learn more about ASTS by visiting our website at ASTS.org.
從這批敬業的小團體發展壯大，我們現在代表着2000多名致力於通過器官捐獻和移植手術拯救和改善生命的移植專業人士。了解更多關於ASTS的信息，請訪問我們的網站asts.org。
*Denotes Current TTC Members
*標記當前TTC成員
MEDIA CONTACTS:
媒體聯繫人：
Shauna O'Brien – AST: [email protected]
Shauna O'Brien – 醫院: [email protected]
Anna Shults – ASTS: [email protected]
Anna Shults – 醫院S: [email protected]
SOURCE American Society of Transplantation
來源 美國移植協會
