Novartis' LDK378 latest to receive FDA Breakthrough Therapy designation

Swiss drug major Novartis (NOVN: VX) says that its investigational compound LDK378 has received Breakthrough Therapy designation from the US Food and Drug Administration for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who had progressed during treatment with, or were intolerant to, crizotinib.

There are limited treatment options for patients with ALK+ NSCLC, who tend to be non-smokers and younger than NSCLC patients without an ALK translocation, Novartis noted.

Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy Designation is intended to expedite the development and review time for a potential new medicine "to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development." The designation includes all of the fast track program features, as well as more intensive FDA guidance. The Breakthrough designation is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.