[Unmet needs: patients with statin intolerance or familial hypercholesterolemia]

The achievement of low-density lipoprotein (LDL) therapeutic targets is especially difficult in some patients at high cardiovascular risk. These patients include persons with statin intolerance and those with very high LDL cholesterol (LDLc) levels such as persons with familial hypercholesterolemia. The proportion of statin-intolerant patients is between 7% and 29%. Alternative lipid-lowering drugs (including ezetimibe) are less effective and are not free from adverse effects. Both alirocumab, with the ODYSSEY ALTERNATIVE study, and evolocumab, with the GAUSS study, have shown strong lipid-lowering efficacy, with much greater tolerability than currently available alternatives, with the result that a larger number of patients achieve therapeutic targets. In familial hypercholesterolemia, the monogenic metabolic disease most frequently associated with high cardiovascular risk, early intervention is cost-effective. Although statins have substantially improved the prognosis of familial hypercholesterolemia, many affected individuals are far from achieving the recommended therapeutic targets. In this patient group, PCSK9 inhibition with monoclonal antibodies has also been shown to be highly effective in reducing LDLc, especially in heterozygous individuals. The studies performed to date have shown that these drugs are safe and effective and can help many patients with familial hypercholesterolemia to drastically reduce their cardiovascular risk.