Vertex Announces FDA Fast Track Designation And Initiation Of Phase 1/2 Clinical Trial For VX-880 In Type 1 Diabetes
Vertex Announces FDA Fast Track Designation And Initiation Of Phase 1/2 Clinical Trial For VX-880 In Type 1 Diabetes
– VX-880 is the first investigational stem cell-derived therapy utilizing fully differentiated, insulin-producing pancreatic islet cells for the treatment of type 1 diabetes –
– VX-880是第一个研究中的干细胞衍生疗法，利用完全分化的、产生胰岛素的胰岛细胞治疗1型糖尿病。
– VX-880 is the first and only pancreatic islet replacement therapy known to receive Fast Track Designation –
– VX-880是已知的第一种也是唯一一种获得Fast Track称号的胰岛替代疗法-
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for VX-880 and that the company has initiated a clinical trial for VX-880 in patients who have type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness.
Vertex制药公司(纳斯达克：VRTX)今天宣布，美国食品和药物管理局(FDA)已授予VX-880快速通道称号，该公司已启动VX-880在患有严重低血糖和降糖意识受损的1型糖尿病(T1D)患者中的临床试验。
"This program has its roots in the groundbreaking work that began in Dr. Doug Melton's lab, progressed at Semma Therapeutics, and has been accelerated and brought to the clinic by the team at Vertex," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. "Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets. We are very pleased to have received FDA's Fast Track Designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients."
Vertex公司执行副总裁兼细胞和遗传疗法首席执行官巴斯蒂亚诺·桑纳博士说：“这项计划源于道格·梅尔顿博士的实验室开始的开创性工作，在Semma治疗公司取得进展，并由Vertex公司的团队加速并引入临床。”我们的方法是生产完全分化和全功能的胰岛素分泌胰岛的唯一方法。我们非常高兴获得FDA的快速通道称号，这促进了治疗严重疾病和满足未得到满足的医疗需求的药物的开发和加快审查。我们继续紧急工作，将这种创新疗法带给患者。“
"It's a remarkable time for T1D research efforts worldwide, as this investigational treatment enters the clinic," said Camillo Ricordi, M.D., Professor of Surgery, Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami Miller School of Medicine, and Steering Committee Chair for the VX-880 clinical trial. "The field's experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D."
医学博士、迈阿密大学米勒医学院糖尿病研究所(DRI)和细胞移植中心主任、VX-880临床试验指导委员会主席、外科教授、医学博士卡米洛·里奥迪说：“随着这种研究疗法进入临床，这是全世界T1D研究工作的非凡时刻。”卡米洛·里奥迪是迈阿密大学米勒医学院糖尿病研究所(DRI)和细胞移植中心的主任，他是医学博士，也是糖尿病研究所(DRI)和迈阿密大学米勒医学院细胞移植中心的主任。该领域在有限的身体胰岛移植方面的经验，其中一些患者已经经历了多年的胰岛素长期独立，为细胞疗法对患有T1D的患者带来变革的潜力提供了重要的概念证明。“
The first clinical trial sites at the University of Miami Health System, the University of Pennsylvania and Massachusetts General Hospital are open for enrollment, and additional sites will be activated this year. To learn more visit clinicaltrials.gov.
迈阿密大学卫生系统(University Of Miami Health System)、宾夕法尼亚大学(University Of Pennsylvania)和马萨诸塞州总医院(Massachusetts General Hospital)的首批临床试验地点已经开放注册，今年还将启动更多地点。要了解更多信息，请访问Clinicaltrials.gov.
译文内容由第三方软件翻译。