拜耳多吉美（Nexavar）新适应症用于分化型甲状腺癌获FDA批准

拜耳（Bayer）和Onyx制药11月22日宣布，口服多激酶抑制剂多吉美（Nexavar，通用名：sorafenib，索拉非尼）已获FDA批准，用于局部晚期或转移性放射性碘(RAI)难治性分化型甲状腺癌的治疗。

Nexavar是首个获FDA批准专门用于放射性碘（RAI）难治性分化型甲状腺癌的药物。

Nexavar新适应症的获批，是基于III期DECISION临床试验的数据，结果表明，与安慰剂相比，sorafenib显著延长了患者的疾病无进展生存期（PFS））（10.8个月 vs 5.8个月，p＜0.0001），达到了研究的主要终点。

今年7月，拜耳和Onyx制药分别向欧洲药品管理局（EMA）和FDA提交了Nexavar用于治疗局部晚期或转移性放射性碘（RAI）难治性分化型甲状腺癌治疗的上市许可申请（MA）和补充新药申请（sNDA），同时，FDA于今年8月授予了Nexavar该适应症sNDA优先审查资格。

关于多吉美（Nexavar）：

Nexavar是一种口服抗癌药物，目前已获全球100多个国家批准，用于肝癌和晚期肾癌的治疗。在欧洲，Nexavar获批用于肝细胞癌（HCC）及既往经α干扰素或白介素2（IL-2）治疗失败或被认为不适用这类疗法的晚期肾细胞癌（RCC）的治疗。

在临床前研究中，Nexavar已被证明能够抑制参与细胞增殖和血管生成过程中的多种激酶，包括Raf激酶、VEGFR-1、VEGFR-2、VEGFR-3、PDGFR-B、KIT、FLT-3和RET。

目前，拜耳和Onyx制药、国际研究团体、政府机构及个体研究者也在调查Nexavar在其他癌症中的疗效。

英文原文：Nexavar® Receives Approval for the Treatment of Differentiated Thyroid Cancer in the U.S.

First and only FDA-approved treatment option for patients with this type of thyroid cancer / FDA approval based on data from Phase III DECISION trial, in which sorafenib significantly extended progression-free survival compared to placebo
Berlin, November 22, 2013 – Bayer HealthCare and Onyx Pharmaceuticals, Inc., an Amgen subsidiary (Nasdaq: AMGN), today announced that the U.S. Food and Drug Administration (FDA) has approved the oral multi-kinase inhibitor Nexavar® (sorafenib) for the treatment of patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma (DTC) that is refractory to radioactive iodine (RAI) treatment. Nexavar was approved following a priority review by the FDA, a designation reserved for drugs that offer a significant improvement in treatment over existing options.

“Nexavar is an important treatment option for patients with certain kinds of cancers, and this latest indication for RAI-refractory differentiated thyroid cancer addresses a serious unmet medical need,” said Kemal Malik, M.D., Member of the Bayer HealthCare Executive Committee and Head of Global Development. “Bayer is committed to exploring the full clinical potential of our therapies to make a difference in the lives of patients and physicians.”

“In the DECISION trial, patients with this type of advanced thyroid cancer who were treated with sorafenib nearly doubled their time to disease progression or death compared to patients treated with placebo,” said Martin Schlumberger, M.D., of Institut Gustave-Roussy in Villejuif, France and co-lead investigator of the DECISION trial. “We are pleased that patients in the United States now have a new and noteworthy treatment option.”

DECISION Trial Design
The FDA approval is based on data from the Phase III DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial, an international, multicenter, placebo-controlled study. A total of 417 patients with locally advanced or metastatic, progressive, RAI-refractory, differentiated thyroid cancer (papillary, follicular, Hürthle cell and poorly differentiated) who had received no prior chemotherapy, tyrosine kinase inhibitors, monoclonal antibodies that target VEGF or VEGF receptor, or other targeted agents for thyroid cancer were randomized to receive 400 mg of oral sorafenib twice daily (207 patients) or matching placebo (210 patients). Ninety-six percent of randomized patients had metastatic disease.

In the trial, sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study, compared to placebo (HR=0.59 [95% CI, 0.46-0.76]; p<0.001), which represents a 41 percent reduction in the risk of disease progression or death for patients who received sorafenib compared to placebo-treated patients. The median PFS was 10.8 months in patients treated with sorafenib, compared to 5.8 months in patients receiving placebo.

The safety and tolerability profile of sorafenib in patients in the trial was generally consistent with the known profile of sorafenib. The most common treatment-emergent adverse events in the sorafenib arm were hand-foot skin reaction, diarrhea, alopecia, weight loss, fatigue, hypertension and rash. Results from the trial were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) in June 2013.

About Thyroid Cancer
Thyroid cancer is the most common endocrine malignancy. There are more than 213,000 new cases of thyroid cancer annually and approximately 35,000 people die from thyroid cancer worldwide each year.

Papillary, follicular (including Hürthle cell) and poorly differentiated types of thyroid cancer are classified as “differentiated thyroid cancer” and account for approximately 94 percent of all thyroid cancers. While the majority of differentiated thyroid cancers are treatable, RAI-refractory locally advanced or metastatic disease is more difficult to treat and is associated with a lower patient survival rate.

About Nexavar® (sorafenib)
Nexavar®, an oral anti-cancer therapy for liver cancer and for the treatment of patients with advanced kidney cancer, is currently approved in more than 100 countries worldwide. In Europe, Nexavar is approved for the treatment of hepatocellular carcinoma (HCC) and for the treatment of patients with advanced renal cell carcinoma (RCC) who have failed prior interferon-alpha or interleukin-2 based therapy or are considered unsuitable for such therapy.

In preclinical studies, Nexavar has been shown to inhibit multiple kinases thought to be involved in both cell proliferation (growth) and angiogenesis (blood supply) – two important processes that enable cancer growth. These kinases included Raf kinase, VEGFR-1, VEGFR-2, VEGFR-3, PDGFR-B, KIT, FLT-3 and RET.

Nexavar is also being evaluated by Bayer and Onyx, international study groups, government agencies and individual investigators in a range of other cancers.

Nexavar is co-developed by Onyx and Bayer, except in Japan where Bayer manages all development. The companies co-promote Nexavar in the U.S. Outside of the U.S. Bayer has exclusive marketing rights, and Bayer and Onyx share profits globally, excluding Japan.