ROS1-positive NSCLC occurs in approximately 1% of NSCLC cases and it represents a particular molecular subgroup of NSCLC.
The breakthrough therapy designation was based on a data from an expansion cohort of a global Phase I trial (Study 1001), which evaluated Xalkori in 50 patients with ROS1-positive advanced NSCLC.
Results from this trial showed that Xalkori exhibited marked anti-tumor activity in patients with ROS1-positive advanced NSCLC.
Pfizer Oncology Clinical Development and Medical Affairs senior vice-president and chief medical officer Dr Mace Rothenberg said: "We are excited that the FDA has granted Breakthrough Therapy designation for Xalkori as a potential treatment for patients with ROS1-positive NSCLC.
"Xalkori pioneered precision medicine for ALK-positive metastatic NSCLC, and ROS1 represents a second molecular subgroup of NSCLC in which Xalkori has demonstrated a level of anti-tumor activity that can potentially make a real difference for patients."
In the US, Xalkori is currently approved to treat patients with metastatic NSCLC whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test.
The company said that it will work closely with the FDA to develop Xalkori for ROS1-positive NSCLC as well as provide the information needed to support a potential regulatory submission.