On November 16, 2015, the U.S. Food and Drug Administration (FDA) granted accelerated approval to osimertinib for the treatment of patients with advanced non-small cell lung cancer (NSCLC). Osimertinib is currently approved for patients with tumors harboring a specific epidermal growth factor receptor (EGFR) mutation (T790M) that have progressed after treatment with other EGFR blockade therapies.
“Our understanding of the molecular basis of lung cancer and why these cancers develop resistance to previous treatments is rapidly evolving,” said Dr. Richard Patzdur, Director of the Office of Hematology and Oncology Products at the FDA’s Center for Drug Evaluation and Research. “This approval provides a new treatment option for patients testing positive for the EGFR resistance mutation (T790M), based on substantial evidence from clinical trials demonstrating that osimertinib is significantly effective in reducing tumor volume in more than half of the patients treated.”
Al, Director of the Office of In Vitro Diagnostics and Radiation Health at the FDA’s Center for Devices and Radiation Health… Dr. Beto Gutierrez stated, “The approval of safe and effective companion diagnostic tests and drugs continues to be a significant advancement in oncology. The advent of cobasEGFR mutation test v2 meets the need to detect this important EGFR gene mutation, which may alter treatment outcomes.”
Clinical Studies of Osimertinib
The safety and efficacy of osimertinib were demonstrated in two multicenter, single-arm studies involving 411 patients with advanced EGFR T790M mutation-positive non-small cell lung cancer whose disease had progressed after EGFR blockade therapy. In the two studies, 57% of patients in the first study and 61% in the second experienced complete or partial tumor shrinkage (referred to as objective response rate). Continued approval for this indication may depend on further confirmatory studies.
Side Effects of Osimertinib
The most common side effects of osimertinib are diarrhea and skin and nail problems, such as dry skin, rash, and infection or redness around the nails. It may cause serious side effects, including lung inflammation and heart damage. It may also harm a developing fetus.
Osimertinib Receives Orphan Drug Designation
The FDA has granted AstraZeneca's osimertinib Breakthrough Therapy Designation, Priority Review Designation, and Orphan Drug Designation. Breakthrough Therapy Designation is granted to drugs intended to treat serious diseases when preliminary clinical evidence at the time of application submission suggests that the drug may be a significant improvement over existing therapies. Priority Review Designation is granted to drug applications that demonstrate a significant improvement in safety or efficacy in the treatment of serious diseases.
