美國制藥巨頭默沙東（Merck &Co）近日收獲“苦澀”的幸福,，曾經(jīng)被FDA取消突破性藥物（BTD）資格的丙肝全口服雞尾酒Grazoprevir/Elbasvir(MK-5172/MK-8742)再次獲得了FDA的2個新的突破性藥物（BTD）資格,，分別為：（1）治療基因型4慢性丙型肝炎病毒（HCV GT4）感染者的BTD資格,；（2）治療伴有終末期腎病正在進行血液透析治療的基因型1 HCV（HCV GT1）感染者的BTD資格。
默沙東表示,，該藥的監(jiān)管計劃仍按原計劃進行，將在2015年上半年向FDA提交新藥申請（NDA）,。業(yè)界認為,，該雞尾酒有望在今年第三季度獲得批準，成為市面上第3個丙肝雞尾酒,，而其競爭對手為吉利德的丙肝雞尾酒Harvoni及艾伯維的丙肝雞尾酒Viekira Pak,，這2個藥物目前已獲美歐2大市場批準上市。
慢性丙型肝炎（HCV）一直是默沙東研發(fā)的重心,，丙肝雞尾酒Grazoprevir/Elbasvir對該公司而言意義重大,。之前，F(xiàn)DA曾在2013年10月授予丙肝雞尾酒Grazoprevir/Elbasvir治療基因型1 HCV（HCV GT1）感染者的BTD資格,，但在2015年2月初卻撤銷了,，一同被取消BTD資格的還有百時美施貴寶的丙肝藥物Daclatasvir。FDA表示,，撤銷這2個丙肝藥物BTD資格是因為當前丙肝治療領域的迅速發(fā)展,。而FDA這一決定，對默沙東而言無疑是個重大打擊,。
FDA的突破性療法認定（BTD）旨在加速開發(fā)及審查治療嚴重的或威脅生命的疾病的新藥,。獲得BTD資格的藥物,，在研發(fā)時能得到包括FDA高層官員在內(nèi)的更加密切的指導，保障在最短時間內(nèi)為患者提供新的治療選擇,。此次,，F(xiàn)DA重新授予默沙東丙肝雞尾酒Grazoprevir/Elbasvir 2個新的BTD資格，反映了HCV GT1和HCV GT4治療領域迫切的醫(yī)療需求,，同時也是對該雞尾酒在丙肝臨床治療中相對現(xiàn)有療法取得實質(zhì)性改善作用的積極肯定,。
Grazoprevir/Elbasvir是一種實驗性、每日一次的復方單片,，由grazoprevir（一種NS3/4A蛋白酶抑制劑）和elbasvir（一種NS5A復制復合體抑制劑）組成,。作為默沙東廣泛臨床試驗項目的一部分，Grazoprevir/Elbasvir目前正被調(diào)查用于數(shù)個基因型HCV感染及難治性丙肝群體的治療,，包括HIV/HCV合并感染,、晚期慢性腎臟病、遺傳性血液疾病,、肝硬化以及正在接受阿片類藥物替代治療的丙肝群體,。
英文原文：Grazoprevir/Elbasvir, Merck’s Investigational Chronic Hepatitis C Therapy, Granted FDA Breakthrough Therapy Designations; New Phase 2 and 3 Data in Multiple HCV Patient Types to be Presented at The International Liver CongressTM 2015
Congress Highlights Include Results from Trials in a Wide Range of HCV Patients -- Patients with Chronic Kidney Disease, HIV Co-infection, Cirrhosis, and Prior Treatment Failures
Company Remains on Track for NDA Filing with the U.S. FDA During First Half of 2015
KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE:MRK), known as MSD outside of the United States and Canada, today announced that grazoprevir/elbasvir, an investigational single tablet regimen for the treatment of chronic hepatitis C virus (HCV) infection, has received two new Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA) for the treatment of patients with chronic HCV genotype 4 (GT4) infection, and for the treatment of chronic HCV genotype 1 (GT1) infection in patients with end stage renal disease on hemodialysis.
“HCV remains a global public health epidemic. At Merck, we are focused on the development of an efficacious, well-tolerated, once-daily therapy that can be used to treat multiple genotypes and a diverse population of chronic HCV patients,” said Dr. Eliav Barr, vice president, infectious diseases, Merck Research Laboratories. “Our clinical program is among the largest and most comprehensive, with studies dedicated to patient populations where significant unmet medical need still exists, such as prior treatment failures, as well as those living with co-morbid conditions, including HIV infection, chronic kidney disease and individuals on opiate substitution therapy.”
In October 2013, the FDA granted Breakthrough Therapy designation for grazoprevir/elbasvir for the treatment of patients with chronic HCV genotype 1 (GT1). In January 2015, the FDA notified Merck of its intention to rescind that Breakthrough Therapy designation. The FDA has now granted two new Breakthrough Therapy designations for grazoprevir/elbasvir; the designations are now for the treatment of patients infected with chronic HCV GT1 with end stage renal disease on hemodialysis, and patients infected with chronic HCV genotype 4 (GT4). Breakthrough Therapy designation is intended to expedite the development and review of a candidate that is planned for use, alone or in combination, to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
Grazoprevir/elbasvir is an investigational, once-daily single tablet regimen consisting of grazoprevir (NS3/4A protease inhibitor) and elbasvir (NS5A replication complex inhibitor). As part of Merck’s broad clinical trials program, grazoprevir/elbasvir is being studied in multiple HCV genotypes and in patients with difficult-to-treat conditions such as HIV/HCV co-infection, advanced chronic kidney disease, inherited blood disorders, cirrhosis and those on opiate substitution therapy.
Merck’s Commitment to HCV
For nearly 30 years, Merck has been at the forefront of the response to the HCV epidemic. Merck employees are dedicated to applying their scientific expertise, resources and global reach to deliver innovative healthcare solutions that support people living with HCV worldwide.
