--诺华（Novartis）今天公布了抗癌药afinitor（依维莫司，everolimus）的一个全球性III期试验的结果，该项试验在对索拉非尼（sorafenib）不耐受或经sorafenib治疗后病情恶化的局部晚期或转移性肝癌（HCC）患者中开展，与安慰剂相比，afinitor未能延长总生存期。诺华将不再推进afinitor用于HCC适应症的监管申请。
到目前为止，afinitor已被证明对许多类型的肿瘤有效，包括激素受体阳性的晚期乳腺癌、晚期胰腺癌、神经内分泌肿瘤及晚期肾细胞癌。
该项肝癌试验的数据，不影响全球范围内afinitor用于这些适应症的监管批准。
目前，afinitor也处于III期开发用于其他疾病，包括：胃肠道和肺神经内分泌肿瘤（NET）、HER2阳性乳腺癌、淋巴瘤、结节性硬化症（TSC）。这些实验的结果，预计将于2014年和2015年获得。
英文原文：Novartis study of Afinitor? in advanced liver cancer does not meet primary endpoint of overall survival
*Everolimus did not show survival benefit for patients with advanced hepatocellular carcinoma (HCC) after progression on or intolerance to sorafinib[1]
*Study results do not impact use of Afinitor in other indications approved by health authorities around the world
*Novartis continues to study Afinitor in other diseases with high unmet needs including GI and lung NET, HER2 positive breast cancer, lymphoma and TSC
Basel, August 7, 2013 - Novartis announced today that results of a global Phase III study showed that Afinitor? (everolimus) did not extend overall survival compared to placebo in patients with locally advanced or metastatic hepatocellular carcinoma (HCC) after progression on or intolerance to sorafenib[1]. Hepatocellular carcinoma, an aggressive and debilitating cancer, is the most common type of liver cancer[2].Novartis will not proceed with regulatory filings of Afinitor in this indication.
"While we are disappointed with these results, Novartis remains committed to studying everolimus through a robust research and development program to address unmet needs in different types of cancer," said Alessandro Riva, Global Head, Oncology Development & Medical Affairs, Novartis Oncology. "To date, Afinitor has proven efficacy in a number of tumor types, including hormone receptor positive advanced breast cancer, advanced pancreatic neuroendocrine tumors and advanced renal cell carcinoma."
The results of the HCC trial do not impact the worldwide approvals of Afinitor for these other indications. Everolimus is also in Phase III development in other diseases, including gastrointestinal and lung neuroendocrine tumors (NET), HER2 positive breast cancer, lymphoma and tuberous sclerosis complex (TSC). Results of these trials are expected during 2014 and 2015.
Study details
The Phase III study, EVOLVE-1 (EVerOlimus for LiVer cancer Evaluation-1), is a randomized, double-blind, placebo-controlled trial examining the efficacy and safety of everolimus versus placebo, plus best supportive care (BSC), in adult patients with advanced HCC whose disease progressed after treatment with or who were intolerant to sorafenib, a targeted therapy[3]. The study results continue to be evaluated and will be presented at an upcoming medical conference.
EVOLVE-1 involved 546 patients and was conducted at 156 sites worldwide. Patients in the trial were randomized (2:1) to receive therapy with everolimus 7.5 mg/day orally plus BSC or placebo plus BSC. The primary endpoint was overall survival. Secondary endpoints included time to tumor progression, disease control rate,time to deterioration of performance status, safety and quality of life