June 15, 2021—Canada’s medical regulatory agency approved the use of selpercatinib monotherapy for treatment of patients with metastatic RET fusion–positive NSCLC. Health Canada based its conditional approval on the LIBRETTO-001 phase I/11 trial, with two phase III trials pending to confirm clinical benefit.
LIBRETTO-001, a multicenter, open-label, multicohort trial, enrolled 144 patients with advanced RET fusion–positive NSCLC. The overall response rate (ORR) for the 105 patients who had previously received treatment was 64% (95% CI: 54%, 73%), with a median duration of response (DoR) of 17.5 months. Of the 39 patients who had not received prior systemic treatment, ORR was 85% (95% CI: 70%, 94%; DoR not reported). Out of 11 patients with measurable brain metastases who were previously treated, 10 were observed to have intracranial responses.
The most common adverse reactions experienced by at least 25% of patients included but were not limited to elevations in hepatic transaminases, decreased lymphocyte counts, and dry mouth.