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是第一個研究中的幹細胞衍生療法，利用完全分化的、產生胰島素的胰島細胞治療1型糖尿病。
– VX-880 is the first and only pancreatic islet replacement therapy known to receive Fast Track Designation –
– VX-880是已知的第一種也是唯一一種獲得Fast Track稱號的胰島替代療法-
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製藥公司(納斯達克：VRTX)今天宣佈，美國食品和藥物管理局(FDA)已授予VX-880快速通道稱號，該公司已啟動VX-880在患有嚴重低血糖和降糖意識受損的1型糖尿病(T1D)患者中的臨牀試驗。
"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公司執行副總裁兼細胞和遺傳療法首席執行官巴斯蒂亞諾·桑納博士説：“這項計劃源於道格·梅爾頓博士的實驗室開始的開創性工作，在Semma治療公司取得進展，並由Vertex公司的團隊加速並引入臨牀。”我們的方法是生產完全分化和全功能的胰島素分泌胰島的唯一方法。我們非常高興獲得FDA的快速通道稱號，這促進了治療嚴重疾病和滿足未得到滿足的醫療需求的藥物的開發和加快審查。我們繼續緊急工作，將這種創新療法帶給患者。“
"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."
醫學博士、邁阿密大學米勒醫學院糖尿病研究所(DRI)和細胞移植中心主任、VX-880臨牀試驗指導委員會主席、外科教授、醫學博士卡米洛·里奧迪説：“隨着這種研究療法進入臨牀，這是全世界T1D研究工作的非凡時刻。”卡米洛·里奧迪是邁阿密大學米勒醫學院糖尿病研究所(DRI)和細胞移植中心的主任，他是醫學博士，也是糖尿病研究所(DRI)和邁阿密大學米勒醫學院細胞移植中心的主任。該領域在有限的身體胰島移植方面的經驗，其中一些患者已經經歷了多年的胰島素長期獨立，為細胞療法對患有T1D的患者帶來變革的潛力提供了重要的概念證明。“
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.
邁阿密大學衞生系統(University Of Miami Health System)、賓夕法尼亞大學(University Of Pennsylvania)和馬薩諸塞州總醫院(Massachusetts General Hospital)的首批臨牀試驗地點已經開放註冊，今年還將啟動更多地點。要了解更多信息，請訪問Clinicaltrials.gov.
譯文內容由第三人軟體翻譯。