First Oral Non-Statin LDL-C Lowering Drug Approved in 2 Decades

February 25, 2020

Bempedoic acid is the first oral, once-daily, non-statin cholesterol lowering medication to receive FDA approval since 2002.

The US Food and Drug Administration (FDA) approved bempedoic acid (Nexletol, Esperion) a once-daily, oral, non-statin LDL-cholesterol (LDL-C) lowering tablet for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL-C.

Bempedoic acid-indicated as an adjunct to diet and maximally tolerated statin therapy-received approval on February 21, 2020.

“The FDA approval of Nexletol provides an important option for patients living with elevated LDL-C and ASCVD or increased risk for cardiovascular disease because of HeFH,” said Christie M. Ballantyne, MD, chairman of Esperion’s Phase 3 Executive Committee in a company press release. “There are millions of patients who are unable to reach their LDL-C targets despite available medicines. Nexletol is the first oral, once-daily, non-statin treatment option for indicated patients in nearly two decades.”

Bempedoic acid is an oral adenosine triphosphate citrate lyase inhibitor that lowers LDL-C by reducing cholesterol and fatty acid synthesis in the liver.

“There are millions of patients who are unable to reach their LDL-C targets despite available medicines. Nexletol is the first oral, once-daily, non-statin treatment option for indicated patients in nearly two decades.”

The approval is based on data from several pivotal global phase 3 clinical trials, the most recent being CLEAR Wisdom which evaluated the long-term safety, tolerability, and efficacy of bempedoic acid 180 mg vs placebo in >750 patients with HeFH or ASCVD already on maximally tolerated statin therapy. Results of CLEAR Wisdom-published November 12, 2019 in JAMA-showed a 17% decrease in LDL-C with bempedoic acid vs placebo after 12 weeks.

A few months earlier, results from the randomized, controlled CLEAR Harmony study were published in The New England Journal of Medicine. In the safety study that examined 2230 patients, bempedoic acid was shown to reduce mean LDL-C by 16.5%, however, it was linked to more adverse events leading to discontinuation and a higher incidence of gout.

The effect of bempedoic acid on cardiovascular morbidity and mortality have not been determined, the company press release noted.

The most common adverse reactions (incidence ≥2% and greater vs placebo) reported with bempedoic acid use include upper respiratory tract infection, muscle spasms, hyperuricemia, back pain, abdominal pain or discomfort, bronchitis, pain in extremity, anemia, and elevated liver enzymes.

“Nexletol 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,” said Tim Mayleben, president and chief executive officer of Esperion in the same press release. “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.”

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