CHMP建议批准吉利德丙肝新药sofosbuvir

吉利德（Gilead）11月22日宣布，丙型肝炎药物Sovaldi（sofosbuvir 400mg 片剂）已获得了欧洲药品管理局（EMA）人用医药产品委员会（CHMP）的积极意见。CHMP建议批准Sovaldi联合其他药物，用于慢性丙型肝炎（HCV）成人感染者的治疗。

Sofosbuvir是一种每日一次的口服核苷类似物聚合酶抑制剂，开发用于慢性丙型肝炎病毒（HCV）感染的治疗。

在欧洲和世界各地，慢性丙型肝炎（HCV）是导致肝癌和肝移植的主要原因。当前的HCV护理标准涉及长达48周的含聚乙二醇干扰素（peg-IFN）/利巴韦林（RBV）方案。这些方案并不总是有效，而且具有显著的副作用，并与其他药物具有用药禁忌。在欧洲，许多患者被认为不适合当前的治疗方案。

CHMP通过加速审批程序对sofosbuvir上市许可申请（MAA）进行了评估，如果获批，Sovaldi有望于2014年第一季度在欧盟上市。sofosbuvir的MAA，由6个III期研究（NEUTRINO, FISSION, POSITRON, FUSION, VALENCE, PHOTON-1）的数据支持。

在美国，FDA顾问委员会于今年10月25日以15:0的投票结果，支持批准sofosbuvir，FDA预计将于2013年12月8日作出审查决定。

如果获批，sofosbuvir将成为用于C型肝炎治疗的首个全口服组合治疗方案中的重要组成部分，并有望消除传统注射药物的需求。

根据汤姆森路透的数据，分析师们平均预测，sofosbuvir在2014年的销售额将达到17.3亿美元。Gilead则估计，sofosbuvir一旦上市，市值将达110亿美元。

英文原文：European CHMP Adopts Positive Opinion for Gilead Sciences’ Sovaldi® for the Treatment of Chronic Hepatitis C Infection

FOSTER CITY, Calif.--(BUSINESS WIRE)--Nov. 22, 2013-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion on the company’s Marketing Authorisation Application (MAA) for Sovaldi® (sofosbuvir 400 mg tablets), an investigational once-daily oral nucleotide analogue polymerase inhibitor for the treatment of chronic hepatitis C virus (HCV) infection in adults. The CHMP opinion supports the approval of Sovaldi for the treatment of HCV in combination with other agents. The CHMP’s recommendation will now be reviewed by the European Commission, which has the authority to approve medicines for use in the 28 countries of the European Union (EU).

Chronic HCV is a major cause of liver cancer and liver transplantation in Europe and around the world. The current standard of care for HCV involves up to 48 weeks of therapy with a pegylated interferon (peg-IFN)/ ribavirin (RBV)-containing regimen. These regimens are not always effective and are associated with significant side effects and contraindications with other medicines. Many HCV patients in Europe are not considered appropriate candidates for current treatment options.

The CHMP opinion was adopted following an accelerated review procedure, which is reserved for medicinal products that are expected to be of major public health interest. This assessment does not guarantee marketing authorisation by the European Commission. However, if approved, Sovaldi could be available in the EU in the first quarter of 2014.

The MAA for Sovaldi is supported primarily by data from four Phase 3 studies, NEUTRINO, FISSION, POSITRON and FUSION in which 12 or 16 weeks of Sovaldi-based therapy was found to be superior or non-inferior to currently available treatment options or historical controls, based on the proportion of patients who had a sustained virologic response (were HCV undetectable) 12 weeks after completing therapy (SVR12). Patients who achieve SVR12 are considered cured of HCV. During the European review, data from two additional Phase 3 studies, VALENCE and PHOTON-1 were filed to the MAA. In the VALENCE study, patients with genotype 3 HCV infection were treated with Sovaldi and RBV for 24 weeks. The PHOTON-1 study evaluated Sovaldi and RBV for 12 weeks in patients with genotype 2 HCV infection co-infected with HIV-1 and for 24 weeks in patients with genotypes 1 or 3 HCV co-infected with HIV-1. In all Phase 3 studies of Sovaldi, no viral resistance to the drug was detected among patients who relapsed following completion of therapy.

To date, nearly 3,000 patients have received at least one dose of Sovaldi in Phase 2 or 3 studies. Sovaldi was well tolerated in clinical studies. Adverse events were generally mild and there were few treatment discontinuations due to adverse events. The most common adverse events occurring in at least 10 percent of patients were consistent with the safety profiles of peg-IFN and RBV and included fatigue, headache, nausea, insomnia, dizziness, pruritis (severe itching) and anemia.

In the United States, an expert advisory committee of the U.S. Food and Drug Administration (FDA) voted unanimously (15-0) on October 25, 2013 that the available data support approval of sofosbuvir. A final decision from the FDA is anticipated by December 8, 2013.

Sovaldi is an investigational product and its safety and efficacy have not been established.