诺华正在开发的用于治疗罕见转移性非小细胞肺癌新药LDK378，诺华获得“突破性新药认定”的3个项目之一，另外两项认定分别为罕见病药BYM338（bimagrumab）和心力衰竭治疗药重组型人松弛素-2 seralaxin。

 由于在一项88名患者参与的Ⅰ期临床研究中显示80%的应答率，在2013年3月15日获得FDA“突破性新药认定”。其适用人群为以辉瑞的Xalkori（克唑替尼）治疗未能奏效或不能耐受的患者。诺华马不停蹄，着手进行一项Ⅲ期临床研究。

Novartis races to the FDA with its 'breakthrough' lung cancer drug

Novartis ($NVS) isn't wasting any time with LDK378, its promising in-development lung cancer drug, filing for FDA approval before embarking on a Phase III study for the breakthrough-designated therapy.

With the application, Novartis is swinging for the full potential of the FDA's newfangled breakthrough status, under which the agency can approve drugs based on mid-stage data. And that's just what the company has in mind, believing its non-small cell lung cancer treatment can sway regulators based on Phase II results alone.

The treatment, designed for patients with metastatic non-small cell lung cancer with anaplastic lymphoma kinase mutations, picked up the FDA's coveted breakthrough therapy designation last year after showing an 80% response rate in a Phase I trial on 88 patients with advanced cases of the disease.

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