On Friday, the U.S. Food and Drug Administration (FDA) expanded the use of Xalkori, a drug commercialized by Pfizer, to treat patients with advanced non-small cell lung cancer whose tumors have a rare mutation called ROS-1.
Xalkori is the only FDA-approved treatment for patients with the rare gene alteration, which represents 1 percent of patients with non-small cell lung cancer, according to the press release published by the FDA.
“The expanded use of Xalkori will provide a valuable treatment option for patients with the rare and difficult to treat ROS-1 gene mutation by giving health care practitioners a more personalized way of targeting ROS-1 positive,” said Dr. Richard Pazdur, FDA’s director for cancer drugs, in the statement.
The twice-a-day drug targets specific genes found in certain patients, recently found effective in people with the ROS-1 gene mutation. It was initially approved in 2011 for another subset of lung cancer patients who have an abnormal gene that stimulates tumor growth, as reported by Seattle Times.
But the powerful drug has significant side effects to be aware of like liver problems, life-threatening or fatal inflammation of the lungs, abnormal heartbeats and partial or complete loss of vision in one or both eyes, the FDA said.
Promising results
The FDA approval was based on a study with 50 patients with ROS-1 positive metastatic in non-small cell lung cancer. Patients received Xalkori twice a day to measure its effect on their lung cancer tumors.
In the study, it was measured overall response rate and the percentage of patients who experience positive responses, which included complete or partial shrinkage of their tumors. At the end of the trial, 66 percent of patients showed a positive response to the drug, an effect that lasted a median of 18.3 months.
The recently expanded drug does not come cheap, like many other cancer treatments. It carries a price of $14,336 per month or about $172,000 per year. The amount does not take into account discounts and rebates often negotiated by insurers.
Source: U.S. FDA