拜耳向EMA及FDA提交多吉美治疗甲状腺癌的上市许可申请(MA)

拜耳（Bayer）昨日宣布，已向欧洲药品管理局（EMA）及FDA提交了有关口服多激酶抑制剂多吉美（Nexavar，通用名：sorafenib，索拉非尼）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治疗的上市许可申请（MA）。

相关监管文件的提交，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），达到了主要终点。

目前，还没有专门用于放射性碘(RAI)难治性分化型甲状腺癌的药物获批

英文原文：Bayer AG : Sorafenib (Nexavar®) Submitted to the EMA and FDA for the Treatment of Locally Advanced or Metastatic Radioactive Iodine-Refractory Differentiated Thyroid Cancer

Currently no approved therapies specifically for differentiated thyroid cancer refractory to radioactive iodine / Regulatory submissions based on data from Phase III DECISION trial, in which sorafenib significantly extended progression-free survival compared to placebo
Berlin, July 1, 2013 - Bayer HealthCare and Onyx Pharmaceuticals, Inc. today announced the submission of applications for marketing authorization in Europe and the U.S. for the oral multi-kinase inhibitor Nexavar® (sorafenib) tablets, for the treatment of locally advanced or metastatic differentiated thyroid cancer refractory to radioactive iodine (RAI).

"The filings in the U.S. and Europe for sorafenib for the potential treatment of differentiated thyroid cancer bring us closer to addressing a significant medical need for patients whose disease has advanced and who have limited or no treatment options," said Kemal Malik, MD, Member of the Bayer HealthCare Executive Committee and Head of Global Development. "Nexavar is already approved in other indications like hepatocellular carcinoma and advanced renal cell carcinoma. We are committed to fully exploring sorafenib's applicability across tumor types, especially in hard-to-treat cancers where there are limited treatment options."

The regulatory submission 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 trial. In the trial, sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study, compared to placebo (HR=0.587 [95% CI, 0.454-0.758]; p<0.0001), which represents a 41 percent reduction in the risk of 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 were 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, rash/desquamation, fatigue, weight loss and hypertension. Results from the trial were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) in June 2013.

DECISION Trial Design
The DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial was an international, multicenter, placebo-controlled study. A total of 417 patients with locally advanced or metastatic, 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.

About Thyroid Cancer
Thyroid cancer has become the fastest-increasing cancer in the world in recent years and is the sixth most common cancer in women. 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, 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.