Familial hypercholesterolemia (FH) is a common genetic disorder that presents 
with robust increases in low-density lipoprotein cholesterol (LDL-C) and can 
lead to premature cardiovascular disease. There are heterozygous and homozygous 
forms. The diagnosis is usually made based on blood cholesterol levels, clinical 
signs and family history. Genetic testing can be used to confirm the diagnosis. 
Effective lowering of LDL-C in FH can prevent cardiovascular morbidity and 
mortality, however, the disease remains greatly underdiagnosed. The mainstay of 
pharmacologic therapy in FH patients is high-dose statins, which are often 
combined with other lipid-lowering agents. The homozygous form is mainly treated 
with lipid apheresis. Guideline-recommended target levels of LDL-C are often not 
reached, making new treatment options desirable. Four classes of newer 
lipid-lowering drugs offer promising advances in treating FH, namely the 
apolipoprotein-B synthesis inhibitors (mipomersen), the microsomal transfer 
protein inhibitors (lomitapide), the cholesterol ester transfer protein 
inhibitors (anacetrapib, evacetrapib) and the proprotein convertase 
subtilisin/kexin type 9 inhibitors (evolocumab, alirocumab). In this review, the 
available evidence regarding the use of these drugs in patients with FH is 
discussed, with particular focus on their efficacy and safety.