Bempedoic Acid (ETC-1002) ATP Citrate Lyase Inhibitor Review of a First-in-Class Medication with Potential Benefit in Statin-Refractory CasesZagelbaum, Nicole K., DO, MPH*; Yandrapalli, Srikanth, MD*; Nabors, Christopher, MD, PhD*; Frishman, William H., MD*,†Cardiology in Review: January/February 2019 - Volume 27 - Issue 1 - p 49–56 doi: 10.1097/CRD.0000000000000218 New Therapy Update Abstract Author InformationAuthors Article MetricsMetrics Bempedoic acid (BA; ETC-1002) is a new agent that reduces cholesterol synthesis through inhibition of adenosine triphosphate citrate lyase, an enzyme upstream from 3-hydroxy-3-methylglutaryl-coenzyme A. In animal models, BA also influences fatty acid synthesis, but in humans, its role is limited primarily to lowering low-density lipoprotein cholesterol (LDL-C). In early clinical trials, BA was well tolerated and without major side effects. Alone or in various combinations with atorvastatin and/or ezetimibe, LDL-C lowering ranged from 17% to 64%. In addition, BA lowers levels of non–high-density lipoprotein cholesterol, C-reactive protein, and apolipoprotein B. Statins are first-line agents for primary and secondary prevention of cardiovascular disease. However, muscle-related side effects and other problems such as elevated liver enzymes may limit their use. In addition, LDL-C lowering beyond that provided by statin therapy alone may be needed. BA may be useful in either of these scenarios, as it is relatively free of muscle-related side effects and appears to enhance LDL-C lowering beyond that achieved with statin monotherapy. Phase 3 trials and one outcomes study are currently under way to better define this agent’s potential clinical role. From the *Department of Medicine †Division of Cardiology, New York Medical College at Westchester Medical Center, Valhalla, NY. Disclosure: The authors declare no conflict of interest. Correspondence: Nicole K. Zagelbaum, MD, Department of Medicine, New York Medical College at Westchester Medical Center, 100 Woods Road, Valhalla, NY 10595. E-mail: NicoleZagelbaum@gmail.com. Copyright © 2019 Wolters Kluwer Health, Inc. All rights reserved.