PCSK9 and familial hypercholesterolemia: In the United States, PCSK9 inhibitors are Food and Drug Administration-approved for a range of indications including familial hypercholesterolemia (both heterozygous and homozygous forms), clinical atherosclerotic cardiovascular disease and as adjuncts in patients unable to reach LDL-C targets despite maximally tolerated statins and ezetimibe.