The final overall survival (OS) analysis of data from the phase III EXAM trial reveal no siginificant improvement in OS with the tyrosine-kinase inhibitor cabozantinib, relative to placebo, in unselected patients with progressive medullary thyroid cancer. However, in an exploratory analysis, selected patients with tumours harbouring the RETM918Tmutation had a significant improvement in median OS in response to cabozantinib (44.3 months, versus 18.9 months in patients receiving placebo; P = 0.03), indicating that cabozantinib is an effective treatment of patients with RETM918T-positive disease.