Axitinib, a tyrosine kinase inhibitor (TKI) of the vascular endothelial growth factor receptor (VEGFR), is currently under review for marketing authorization for the second-line therapy of advanced renal cell carcinoma (RCC) by the US Food and Drug Administration (FDA) and received orphan drug designation for this indication by the European Medicines Agency (EMA) in February 2011. One phase III trial (AXIS trial) comparing the efficacy and safety of axitinib to sorafenib, another TKI, was found for this indication. Improved outcomes for progression free survival (PFS) and response rates were shown in the axitinib group, but no significant difference in overall survival (OS). Serious AEs were more frequent in the axitinib group, however, treatment discontinuations were more frequent in the sorafenib group.
The AXIS trial was the first head-to-head comparison in a phase III trial of targeted agents in the treatment of advanced RCC. For this indication, there are currently six different targeted agents approved.