FDA approves bempedoic acid to treat familial hypercholesterolemia

The FDA has approved bempedoic acid as an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH).

The drug, sold as Nexletol by Michigan-based manufacturer Esperion, is reportedly the first oral, once-daily, non-statin LDL-cholesterol-lowering medicine approved in the U.S. for indicated patients since 2002. It’s considered a first-in-class ATP citrate lyase (ACL) inhibitor and works by inhibiting cholesterol synthesis in the liver, thereby lowering a person’s LDL-C.

Even with statins and standard-of-care treatment, it’s estimated that some 15 million patients in the U.S. struggle to achieve guideline-recommended LDL-C levels.

“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 atherosclerotic CVD or HeFH,” Tim Mayleben, president and CEO of Esperion, said in a company 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.”

The effect of Nexletol on cardiovascular morbidity and mortality hasn’t yet been determined, according to the statement; instead, the FDA’s Feb. 21 approval was supported by a global Phase 3 LDL-C-lowering program. According to Esperion, bempedoic acid provided patients an average of 18% placebo-corrected LDL-C lowering when used in conjunction with moderate- or high-intensity statins.

The drug has been “generally well-tolerated” in clinical studies, the company said, though label warnings include hyperuricemia and an increased risk of tendon rupture or injury. Adverse events reported most often in the Phase 3 studies included respiratory tract infections, muscle spasms, hyperuricemia, back pain, abdominal pain, bronchitis, anemia and elevated liver enzymes, but those events were reported similarly in patients who received the placebo drug.

Esperion said it’s working with healthcare providers to offer “broad insurance coverage” for Nexletol, which could cost as little as $10 per fill for eligible patients with commercial insurance. 

“Esperion is committed to achieving the lowest-branded tier coverage for Medicare patients,” the company’s statement read. “Esperion will provide resources to patients whose physician recommends treatment with Nexletol...These resources include educational materials, a dedicated call center, as well as a copay program for eligible patients.”

Bempedoic acid is slated to hit the commercial market as Nexletol on March 30. It will be available by prescription only.