Ezetimibe, in combination with statin therapy, significantly reduces cardiovascular events in individuals with acute coronary syndrome (ACS) or myocardial infarction (MI), as demonstrated in the IMPROVE-IT trial. Furthermore, ezetimibe has been shown to reduce both LDL-C levels and atherosclerotic plaque volume, as evidenced by the PRECISE-IVUS trial. Additionally, ezetimibe monotherapy has demonstrated benefits in reducing cardiovascular risk in older adults with elevated LDL-C levels, as seen in the EWTOPIA 75 trial.

Ezetimibe is approved for use in combination with statin therapy or as monotherapy to reduce LDL-C levels in adults with primary hyperlipidemia, heterozygous familial hypercholesterolemia (HeFH) patients ≥ 10 years old, and patients with mixed hyperlipidemia (as an adjunct to fibrates and diet). It is also indicated to reduce sitosterol and campesterol in patients ≥ 9 years old with homozygous familial sitosterolemia.

Ezetimibe is generally safe, with common side effects including headache, sore throat, and runny nose. Less common adverse effects may include body aches, back pain, diarrhea, joint pain, fatigue, and hepatotoxicity. Rhabdomyolysis has been reported when used in combination with statins. It is contraindicated in patients with active liver disease and has not been studied during pregnancy and lactation.

Ezetimibe is considered cost-effective, particularly when used in high-income countries and for primary prevention. It has been shown to modestly increase life expectancy and is associated with incremental cost-effectiveness ratios (ICERs) ranging from USD 6971.24 to USD 91,500 per QALY, depending on the population and treatment regimen.