Dabrafenib突破性疗法认定的授予，是基于一项正在开展的II期研究的中期疗效和安全性数据。该项研究在25例携带BRAF V600E突变、且既往接受过至少一次含铂化疗方案的NSCLC患者中开展，研究中将dabrafenib进行口服给药。这些中期数据已提交至2013年美国临床肿瘤学会年会。
肺癌是男性和女性中的第二大常见癌症，是目前癌症相关死亡的主要原因之一。肿瘤生物学中的最新进展已确定了肺癌相关的基因突变，如BRAF蛋白中的突变，这些突变能够驱动非小细胞肺癌（NSCLC）中恶性细胞的生长和肿瘤的增殖。据估计，dabrafenib靶向的BRAF V600E突变，约占NSCLC病例的2.0%。
关于Tafinlar（dabrafenib）：
Tafinlar是葛兰素史克开发的一种抗癌药物，该药已分别于2013年5月和9月获FDA和EMA批准，用于携带BRAF V600E突变的手术不可切除性黑色素瘤或转移性黑色素瘤成人患者的治疗。
dabrafenib为一种激酶抑制剂，靶向于BRAF蛋白，这是机体内一个生物信号通路中的关键元件，该信号通路调节细胞的正常生长和死亡，包括皮肤细胞。
转移性黑色素瘤中，约有一半携带BRAF突变，该异常突变能促使黑色素瘤生长和扩散。
英文原文：Tafinlar® receives FDA Breakthrough Therapy designation for non-small cell lung cancer with BRAF mutation
- Marks GSK’s fourth Breakthrough Therapy designation
Issued: Monday 13 January 2014, London UK
GlaxoSmithKline plc (LSE: GSK) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Tafinlar® (dabrafenib) for treatment of patients with metastatic BRAF V600E mutation-positive non-small cell lung cancer (NSCLC) who have received at least one prior line of platinum-containing chemotherapy. Dabrafenib is not approved or licensed anywhere in the world for use in this treatment setting.
The Breakthrough Therapy designation was based on interim efficacy and safety results from an ongoing Phase II study of dabrafenib administered orally to 25 patients who had NSCLC with the BRAF V600E mutation and who had received at least one previous course of chemotherapy. These interim results were presented at the 2013 American Society for Clinical Oncology Annual Meeting.
Lung cancer is the second most common cancer in both men and women and is by far the leading cause of cancer-related death worldwide. Recent advances in the understanding of tumour biology have identified genetic mutations, such as mutation in the BRAF protein, that can drive malignant cell growth and tumour proliferation in NSCLC. It is estimated that the BRAF V600E mutation targeted by dabrafenib is present in approximately 2.0%.[i], [ii] of patients with NSCLC.
