The US Food and Drug Administration (FDA) has approved Tagrisso (osimertinib; AstraZeneca) for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR tyrosine kinase inhibitor (TKI) therapy. This indication is approved under the FDA’s accelerated approval process based on tumor response rate and duration of response.

The approval was based on two multicenter, single-arm, open-label clinical trials in patients with metastatic EGFR T790M mutation-positive NSCLC who had progressed on prior systemic therapy, including an EGFR TKI (Study 1 and 2).  All patients were required to have EGFR T790M mutation-positive NSCLC as detected by the cobas EGFR Mutation Test v2 (Roche) and received osimertinib 80 mg once daily. The major efficacy outcome measure was objective response rate (ORR) according to RECIST v1.1 as evaluated by a Blinded Independent Central Review (BICR). Duration of response (DOR) was an additional outcome measure.

Study 1 (n=201) showed an ORR of 57% (95% CI: 50%, 64%). In Study 2 (n=210) the ORR was 61% (95% CI: 54%, 68%). The majority (96%) of patients in both trials had ongoing responses at the time of primary analysis and the median DOR had not been reached with duration of ongoing responses ranging from 1.1 to 5.6 months after a median duration of follow-up of 4.2 months (Study 1) and 4.0 months (Study 2).  The dose finding phase of Study 1 (n=63) showed an ORR of 51% and median DOR of 12.4 months.

Tagrisso is an EGFR-TKI, a targeted cancer therapy, designed to inhibit both the activating, sensitizing mutations (EGFRm), and T790M, a genetic mutation responsible to EGFR-TKI treatment resistance. Nearly two-thirds of NSCLC patients who are EGFR mutation-positive and experience disease progression after being treated with an EGFR-TKI develop the T790M resistance mutation, for which there have been limited treatment options.

Tagrisso is supplied as 40mg and 80mg tablets in 30-count bottles.

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