The FDA has granted Accelerated Approval for an oral medication to treat patients with advanced non-small cell lung cancer (NSCLC). Tagrisso (osimertinib) now is approved for patients whose tumors have a specific epidermal growth factor receptor (EGFR) mutation (T790M) and whose disease has gotten worse after treatment with other EGFR-blocking therapy.

Lung cancer is the leading cause of cancer death in the U.S., with an estimated 221,200 new diagnoses and 158,040 deaths in 2015, according to the National Cancer Institute. The most common type of lung cancer, NSCLC, occurs when cancer cells form in the tissues of the lung. The EGFR gene is a protein involved in the growth and spread of cancer cells.

“Our understanding of the molecular basis of lung cancer and reasons these cancers become resistant to prior treatments is rapidly evolving,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval provides a new treatment for patients who test positive for the EGFR resistance mutation, T790M, and is based on substantial evidence from clinical trials that shows Tagrisso had a significant effect on reducing tumor size in over half of patients who were treated.” 

The safety and efficacy of Tagrisso were demonstrated in two multicenter, single-arm studies involving a total of 411 patients with advanced EGFR T790M mutation-positive NSCLC whose disease worsened after treatment with an EGFR-blocking medication. In those two studies, 57% of patients in the first study and 61% of patients in the second study experienced a complete or partial reduction in their tumor size (known as objective response rate).

Continued approval for the indication may be contingent upon further confirmatory studies.

The most common side effects of Tagrisso are diarrhea, skin and nail conditions such as dry skin, rash and infection or redness around the fingernails. Tagrisso may cause serious side effects, including inflammation of the lungs and injury to the heart. It also may cause harm to a developing fetus.

The FDA granted AstraZeneca Breakthrough Therapy designation, Priority Review and Orphan Drug designation for Tagrisso.