Based on results of a clinical trial led by Yale Cancer Center (YCC) researchers, the U.S. Food and Drug Administration (FDA) has approved osimertinib for the treatment of adults with early-stage, non-small cell lung cancer (NSCLC) with EGFR gene mutations, which occurs in about 10 percent of patients. The benefit of osimertinib treatment was so substantial during the ADAURA trial that the independent data monitoring committee recommended early unblinding of the multinational randomized controlled phase III trial to make the data available to everyone and to give investigators time to reassess the benefit to participants.

“Osimertinib has demonstrated an unprecedented disease-free survival (DFS) benefit for early-stage lung cancer patients with EGFR mutations who face high rates of recurrence even after surgery and chemotherapy,” said Roy S. Herbst, MD, PhD, chief of medical oncology at YCC and Smilow Cancer Hospital and principal investigator of the ADAURA trial. “This approval will now provide a much-needed treatment option for patients battling this difficult to treat lung cancer.”

Disease-free survival measures the time from randomization to first sign of cancer recurrence or death. In the trial, patients treated with osimertinib had an 83% reduction in the risk of their cancer returning or death.

Osimertinib, an oral, daily medication, shuts down activity of specific genetic mutations that contribute to NSCLC in a subset of patients with the disease. The drug was first approved for use in EGFR mutation-positive advanced lung cancer in the United States in 2015 and is now approved worldwide for treatment in this setting. The ADAURA trial, conducted worldwide with 682 patients, was the first study of osimertinib in non-metastatic EGFR mutation-positive lung cancer.

“This approval reinforces how critical it is to test all lung cancer patients for EGFR mutations before selecting a treatment plan, regardless of their stage at diagnosis,” Herbst added. “In addition, physicians will likely begin to conduct molecular testing for these earlier stage patients now that there is an FDA-approved adjuvant treatment option available.”

The FDA granted the approval of osimertinib to AstraZeneca. The drug will be sold under the name Tagrisso.