The U.S. Food and Drug Administration (FDA) approved crizotinib (Xalkori), for the treatment of ROS1-positive tumors in patients with metastatic non-small cell lung cancer (NSCLC).1 It is the only drug approved for patients with the ROS1 gene alteration, which occurs in approximately 1% of patients with NSCLC.2

“Lung cancer is difficult to treat, in part, because patients have different mutations, some of which are rare,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

“The expanded use of Xalkori will provide a valuable treatment option for patients with the rare and difficult to treat ROS1 gene mutation by giving health care practitioners a more personalized way of targeting ROS1-positive NSCLC.”2

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Crizotinib works by blocking the activity of the ROS1 protein in tumors with the ROS1 gene alteration. The blockage may prevent NSCLC from growing and spreading.

The approval was based on the results of a multicenter, single-arm trial that included 50 patients with metastatic ROS1 rearrangement-positive NSCLC. All received crizotinib 250 mg orally, twice daily.1,2 Efficacy was measured by overall response rate and duration of response.

Results showed that 66% of patients experienced complete or partial tumor shrinkage, which lasted a median of 18.3 months.2

Safety results were consistent with the safety profile evaluated in 1669 patients with anaplastic lymphoma kinase (ALK)-positive metastatic NSCLC.1

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The most common adverse events associated with crizotinib included vision disorders, nausea, diarrhea, vomiting, edema, constipation, elevated transaminases, fatigue, decreased appetite, upper respiratory infection, dizziness, and neuropathy. Serious side effects included liver problems, life-threatening or fatal inflammation of the lungs, abnormal heartbeats, and partial or complete loss of vision in 1 or both eyes.2

Crizotinib was approved in 2011 for the treatment of patients with metastatic NSCLC whose tumors are ALK-positive.2


  1. FDA approves crizotinib capsules [news release]. Silver Spring, MD: U.S. Food and Drug Administration; March 11, 2016. ucm490391.htm. Accessed March 11, 2016.
  2. FDA expands use of Xalkori to treat rare form of advanced non-small cell lung cancer [news release]. Silver Spring, MD: U.S. Food and Drug Administration; March 11, 2016. Accessed March 11, 2016.