Long-term follow-up of selpercatinib for RET-activated thyroid cancer: multinational phase 1-2 trial

LIBRETTO-001 is a multinational phase 1-2 registrational trial of selpercatinib in patients with RET-activated cancers. Long-term follow-up results of selpercatinib in patients with RET-mutant medullary thyroid cancer (MTC) and patients with RET fusion-positive thyroid cancer of different histological subtypes (TC) have now been published. Objective response rate was 82.5% among patients with cabozantinib/vandetanib-naïve MTC and 95.8% among patients with treatment-naïve TC. The figure shows the progression-free survival in the various groups. The safety profile of selpercatinib was consistent with previous reports. The authors conclude that with over 40 months of median follow-up, selpercatinib continued to provide durable and robust responses in treatment-naïve and previously treated RET-activated thyroid cancer.