The FDA has approved the use of osimertinib as a treatment for patients with early-stage epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC).
According to a release from the manufacturer, the approval is for patients who have had tumor resection with curative intent and was made under the Real-Time Oncology Review pilot program. Osimertinib is also approved for patients whose tumors have exon 19 deletions or exon 21 L858R mutations, the release noted.
The FDA based its approval on findings from the phase III ADAURA trial, which showed that osimertinib demonstrated significant improvement in disease-free survival of patients with stage II and IIIA EGFRm NSCLC, and in the overall trial population of patients with stage IB-IIIA disease, which the release noted was a key secondary endpoint.
“Adjuvant [osimertinib] has demonstrated an unprecedented disease-free survival benefit for early-stage lung cancer patients with EGFR mutations who face high rates of recurrence even after successful surgery and subsequent chemotherapy,” said principal investigator Roy S. Herbst, MD, PhD, of the Yale Cancer Center, in the release. “This approval reinforces how critical it is to test all lung cancer patients for EGFR mutations before deciding how to treat them and regardless of their stage at diagnosis.”
This additional testing, according to Herbst, will “help ensure as many patients as possible can benefit from this potentially practice-changing treatment.”
According to the release, adjuvant use of the drug reduced the risk of disease recurrence or death by 83% among patients with stage II and IIIA disease (HR = 0.17; 95% CI, 0.12-0.23; p<0.001). In the overall population of patients with stage IB-IIIA disease, the treatment reduced the risk of disease recurrence or death by 80% (HR 0.20; 95% CI, 0.15-0.27; p<0.001). After two years, the results of the trial showed 89% of patients treated with osimertinib were alive and disease-free, compared to 52% of patients on placebo after surgery. The release noted that the safety and tolerability of osimertinib were similar to what researchers had seen in previous trials in the metastatic setting.
Prior to the approval, the FDA granted osimertinib Breakthrough Therapy Designation in the early-stage disease setting. The release noted that the ADAURA trial was unblinded two years early “based on a determination of overwhelming efficacy.” The release added that the trial is ongoing, with patients continuing to be blinded to treatment. Results of the trial were presented at the recent ASCO Virtual Scientific Program and were published in the New England Journal of Medicine.
The approval process included the FDA and regulatory agencies of five other countries through Project Orbis.