Cholesterol biosynthesis inhibitor gets FDA approval for treatment of high CV risk patients
The first-in-class ATP Citrate Lyase (ACL) inhibitor bempedoic acid was approved by the US Food and Drug Administration (FDA) for the treatment of adults with heterozygous familial hypercholesterolemia or established ASCVD. Bempedoic acid is an oral, once-daily, non-statin LDL-c lowering drug, indicated as an adjunct to diet and maximally tolerated statin therapy.
Bempedoic acid is a first-in-class ACL inhibitor that lowers LDL-c by inhibition of cholesterol synthesis in the liver, resulting in upregulation of LDL receptors. Effects of bempedoic acid on CV morbidity and mortality have not been determined yet. The CV outcomes trial (CLEAR Cardiovascular Outcomes Trial) completed enrollment in August 2019 of 14032 patients with hypercholesterolemia and high CVD risk.
FDA approval of bempedoic acid is supported by data of a phase 3 LDL-c lowering program in more than 3000 patients. In these studies, bempedoic acid resulted in a mean reduction of 18% compared to placebo in background of moderate of high intensity statin.
Bempedoic acid was generally well-tolerated. Warnings and precautions include hyperuricemia (development of gout in some patients), and tendon rupture or injury. Most common adverse events were upper respiratory tract infections, muscle spasms, hyperuricemia, back pain, abdominal pain or discomfort, bronchitis, pain in extremity, anemia and elevated liver enzymes.