FDA Approves New Lipid-Lowering Drug, Bempedoic Acid

(UPDATED) The US Food and Drug Administration has approved bempedoic acid (Nexletol) an oral, once-daily, non-statin LDL cholesterol (LDL-C)-lowering medicine for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL-C, the drug’s maker, Esperion, announced on February 21, 2020.

The drug is the first oral, non-statin, LDL-lowering medication approved since 2002, the company noted.

On February 26, the FDA also cleared a fixed-dose combination containing bempedoic acid and ezetimibe (Nexlizet).

Bempedoic acid has been studied four phase III clinical trials representing more than 3,600 patients. The most recent of these was CLEAR Wisdom, presented at the American College of Cardiology (ACC) 2019 Scientific Session, showing that the addition of bempedoic acid to maximally tolerated statin therapy significantly lowered LDL-C by 15.1% at 12 weeks; it also lowered total cholesterol, apolipoprotein B, non-HDL cholesterol, and C-reactive protein levels. While the study wasn’t powered for clinical events, major adverse cardiovascular events were reduced in the patients treated with bempedoic acid.

Results for CLEAR Harmony, conducted in patients with cardiovascular disease, familial hypercholesterolemia, or both, and LDL-C levels of 70 mg/dL or greater despite taking maximally tolerated statin therapy with or without additional lipid-lowering therapy, had been published just ahead of the ACC meeting. CLEAR Outcomes, the large cardiovascular outcomes trial, is still underway, with results not expected until 2022.  

The novel agent is an inhibitor of ATP citrate lyase, a key enzyme involved in the cholesterol biosynthesis pathway.

“This approval delivers upon a commitment we’ve made to millions of patients for a new treatment alternative if they struggle with bad cholesterol and have ASCVD or HeFH,” Tim Mayleben, president and chief executive officer of Esperion, said in a statement. “Even with maximally tolerated statins, which may mean no statin at all, some of these patients can’t achieve their LDL-C goals. Today’s approval provides them with a new medicine to go along with a healthy diet. We express our sincere gratitude to all of the patients and physicians who put their confidence in Esperion’s team of lipid experts.”

The agent will be commercially available by prescription as of March 30, 2020, a company press release noted.