吉利德（Gilead）4月3日公布了有关丙肝新药Sovaldi（sofosbuvir）的一项III期研究（GS-US-334-0118）的积极数据。该项研究在日本开展，评价了Sovaldi联合利巴韦林（ribavirin，RBV）用于基因型2慢性丙型肝炎病毒（HCV）感染者的治疗。研究数据表明，与预先定义的历史对照持续病毒学应答率（SVR）相比，该项研究达到了主要疗效终点。该项研究中，Sovaldi+RBV治疗组有97%（n=148/153）的患者实现了SVR12（完成治疗后12周的持续病毒学应答），初治患者组和既往治疗组SVR12数据分别为98%（n=88/90）和95%（n=60/63）。接受治疗的153例患者中，11%（n=17）患者存在肝硬化。该项研究的完整数据将提交至未来的科学会议。

吉利德计划于2014年中期向日本的监管机构提交Sovaldi的监管文件。

在工业化国家中，日本有着最高的肝癌发生率，大多数病例由慢性丙型肝炎病毒（HCV）感染所致。在日本，估计有200万丙肝患者，其中约20-30%为基因型2病毒株。在世界各地，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标准护理涉及长达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。

关于

Sofosbuvir为每日一次的口服核苷类似物聚合酶抑制剂，已获FDA、欧盟、加拿大批准，以商品名Sovaldi上市，作为抗病毒治疗方案的一部分，用于慢性丙型肝炎（HCV）的治疗，该药是首个获批可用于C型肝炎全口服治疗方案的药物，在用于特定基因型慢性丙型肝炎治疗时，可消除对传统注射药物干扰素（IFN）的需求。

具体而言，Sovaldi已获批联合利巴韦林（ribavirin）用于基因型2和基因型3慢性丙型肝炎（hepatitis C）成人患者的治疗。同时，Sovaldi已获批联合聚乙二醇干扰素（PEG-IFN）和利巴韦林，用于基因型1和基因型4慢性丙型肝炎初治（treat-naive）成人患者的治疗。

华尔街分析师平均预计，Sovaldi在2017年的销售额将达到91亿美元。在美国，28片/瓶装Sovaldi的批发商采购成本（WAC）费用将为2.8万美元，即每片1000美元，大多数患者需要治疗12周，总费用将高达8.4万美元。

Gilead says Japanese hepatitis C trial meets goal

– Results Confirm Efficacy and Safety of All-Oral Sofosbuvir-Based Regimen for Genotype 2 HCV Patients –

– Japanese Regulatory Filing Planned for Mid-Year –

FOSTER CITY, Calif.--(BUSINESS WIRE)--Apr. 2, 2014-- Gilead Sciences, Inc. (Nasdaq:GILD) today announced topline results from a Phase 3 clinical trial (Study GS-US-334-0118) in Japan eva1uating the once-daily nucleotide analog polymerase inhibitor sofosbuvir in combination with ribavirin (RBV) for the treatment of genotype 2 chronic hepatitis C virus (HCV) infection. The study met its primary efficacy endpoint of superiority compared to a predefined historical control sustained virologic response (SVR) rate. In the study, 97 percent (n=148/153) of genotype 2 HCV-infected patients receiving 12 weeks of an all-oral regimen of sofosbuvir plus RBV achieved a sustained virologic response 12 weeks after completing therapy (SVR12). SVR12 rates among treatment-naïve and treatment-experienced patients were 98 percent (n=88/90) and 95 percent (n=60/63), respectively. Of the 153 patients who received treatment, 11 percent (n=17) had documented cirrhosis.

Japan has one of the highest rates of liver cancer of any industrialized country, and the majority of cases are due to chronic HCV infection. An estimated two million people in Japan are living with HCV infection, and approximately 20-30 percent have the genotype 2 strain of the virus. Current treatment options for genotype 2 HCV infection in Japan involve up to 48 weeks of therapy with pegylated interferon injections, which may not be suitable for certain patients.

In Study GS-US-334-0118, 153 patients (100%) became HCV undetectable by treatment Week 4 and remained undetectable through the remainder of the 12-week treatment period. Post-treatment relapse accounted for five virologic failures. There were no treatment discontinuations due to adverse events and all patients completed the 12 week post-treatment follow-up visit. The most common side effects observed in the study, consistent with the population and safety profile of RBV, included nasopharyngitis, anemia, headache, malaise and pruritis. Full study results will be presented at a future scientific meeting.

“This study confirms the high efficacy of all-oral therapy with sofosbuvir among genotype 2 hepatitis C patients in Japan, regardless of whether they are treatment experienced or new to treatment,” said Norbert Bischofberger, PhD, Executive Vice President of Research and Development and Chief Scientific Officer, Gilead Sciences. “Based on these trial results, Gilead anticipates submitting a New Drug Application for sofosbuvir to the Japanese Pharmaceutical and Medical Devices Agency (PMDA) by mid-2014.”

Gilead established operations in Japan with the formation of Gilead K.K. in Tokyo in September 2012. If approved by the PMDA, sofosbuvir would be the first product to be launched and marketed by Gilead in Japan.

Gilead is also conducting a Phase 3 study in Japan eva1uating the efficacy and safety of a once-daily fixed-dose combination of the NS5A inhibitor ledipasvir 90 mg and sofosbuvir 400 mg with and without ribavirin for the treatment of patients with genotype 1 chronic HCV infection, the most common strain of HCV in Japan. SVR12 results are expected in the second half of 2014.

Sofosbuvir is an investigational product in Japan and its safety and efficacy has not yet been established. The compound has been approved by regulatory authorities in the United States, European unio and Canada and is commercialized under the tradename Sovaldi®. The ledipasvir/sofosbuvir fixed-dose combination is an investigational product and its safety and efficacy has not yet been established.

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