The U.S. Food and Drug Administration has designated an experimental lung cancer drug as a ‘breakthrough therapy,’ which could speed up a new treatment for patients who now have limited options for this life-threatening disease. Drug maker Novartis AG announced on March 15 that its investigational compound LDK378 received the special FDA designation based on early clinical trial data.
LDK378 was tested in phase 1 clinical trials 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 (Pfizer Inc.'s drug Xalkori). Novartis has since launched phase II clinical trials of LDK378 and hopes to start phase III trials later this year.
These newer trials will help establish the safety and efficacy of LDK378. While there’s no guarantee that the experimental drug will ever make it to market, Novartis hopes to begin filing for regulatory approval of LDK378 in early 2014.
The FDA began its new breakthrough therapy program in 2012 to help expedite the approval process for new drugs like LDK378 that represent significant improvements in treatment options for serious or life-threatening conditions. With the usual approval process, it can take as long as 10 years to get a new drug out of the pipeline and into consumer medicine cabinets.
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85 percent of all cases. About 3 to 8 percent of patients with NSCLC have the ALK gene mutation. Treatment options for patients with this gene mutation are very limited. These patients tend to be nonsmokers and younger than NSCLC patients without the ALK mutation.