Am J Health Syst Pharm. 2021 Jan 5;78(2):95-104. doi: 10.1093/ajhp/zxaa352.
PURPOSE: An update on clinical development of a first-in-class oral medication for adjunctive cholesterol lowering in high-risk patients with persistent elevation of low-density lipoprotein cholesterol (LDL-C) despite statin therapy is reviewed.
SUMMARY: Despite the proven efficacy of statin therapy, many patients cannot reach LDL-C goals with statins alone, largely due to inadequate response or intolerance. Nonstatin treatment options to reduce LDL-C include ezetimibe, bile acid sequestrants, and proprotein convertase subtilisin/kexin 9 (PCSK9) inhibitors; however their use has been limited by modest clinical benefit or high treatment costs. Novel nonstatin treatments are in development to further address the needs of this population. Bempedoic acid is a first-in-class oral adenosine triphosphate (ATP) citrate lyase inhibitor being evaluated as an additional treatment option for high-risk patients requiring further reduction in LDL-C. Bempedoic acid has been evaluated in multiple phase 2 and phase 3 trials as monotherapy or for use in combination with ezetimibe and/or statin therapy. Treatment with bempedoic acid has been demonstrated to result in significant reductions in LDL-C and several other cardiovascular risk markers without the myalgia associated with statin therapy.
CONCLUSION: Bempedoic acid, used alone or with ezetimibe in a fixed-dose combination formulation, may be an effective alternative to current guideline-recommended nonstatin therapies in patients who do not attain adequate LDL-C lowering with maximally tolerated statin therapy and in statin-intolerant patients at risk for atherosclerotic cardiovascular disease.