Familial hypercholesterolaemia is a relatively frequently occurring disease that is strongly associated with vascular disease. Current treatment with cholesterol-lowering agents is partly effective but shows variable responses between patients with familial hypercholesterolaemia. Recently, new cholesterol-lowering drugs have been developed. Here we describe the most promising of these new agents for which results from phase 2 or phase 3 trials are available. We will discuss the data regarding lipid-lowering potential and safety issues and speculate about the potential reductions of the residual risk of statin-treated FH patients.