FDA Approves Label Expansions, New Indication for Bempedoic Acid Tablets for CV Prevention
The approval represents the first non-statin treatment indicated to lower low-density lipoprotein for primary prevention patients.
FDA approval stamp
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The FDA approved label expansions for bempedoic acid tablets (Nexletol) and bempedoic acid and ezetimibe tablets (Nexlizet), as well as approved a new indication.1
The label expansions include indications for cardiovascular risk reduction and expanded lowering of low-density lipoprotein (LDL) cholesterol in patients for primary prevention of cardiovascular disease and secondary prevention in patients who have already had a cardiovascular event. The expanded labels support the use of the treatments either with or without statins.
The new indication is for primary hyperlipidemia either with or without a statin, making bempedoic acid and bempedoic acid plus ezetimibe the only non-statin drugs for lowering LDL cholesterol in primary prevention patients.
Bempedoic acid was first approved in 2020 to lower LDL cholesterol in adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease.2 Then in December 2023, the FDA approved a label expansion for bempedoic acid tablet and bempedoic acid and ezetimibe tablet to include treatment of primary hyperlipidemia as a qualifier for existing approved populations.3
“Importantly, these approvals expand the accessibility of our highly effective drugs to primary prevention patients, or to those who are at high risk of having a cardiovascular event, but who have not yet had one,” Sheldon Koenig, president and CEO of Esperion, said in a statement.1 “These approvals also eliminate the statin use requirement, allowing patients to take Nexletol or Nexlizet either with or without a statin, which significantly reduces previously existing prescribing limitations.”
The approvals were based on the data from the phase 3 CLEAR Outcomes trial, double-bind, randomized, placebo-controlled trial of 13,970 patients between the ages of 18 and 85 years. The results were published March 2023 in New England Journal of Medicine.4 Results of the trial were also presented at the 72nd American College of Cardiology (ACC) Scientific Session.5
The study found that after 6 months, the mean LDL cholesterol reduction compared with baseline was greater by 29.2 mg per deciliter with bempedoic acid compared with placebo. The incidence of events making up the primary end point were significantly lower in the treatment arm compared with placebo with events in 11.7% of patients on bempedoic acid compared with 13.3% of patients on placebo (HR 0.87; 95% CI, 0.79-0.96; P = .004).
Bempedoic acid reduced risk of heart attacks 23% with 261 events in the treatment group vs 334 in the placebo (HR 0.77; 95% CI, 0.66-0.91; P = .002). The risk of coronary revascularization was also cut significantly with 435 events in the treatment arm vs 529 events for placebo (HR 0.81; 95% CI, 0.72-0.92; P = .001). There was also a 15% relative risk reduction for MACE-3 (death from a cardiovascular case, nonfatal stroke, or nonfatal myocardial infarction).
The reported adverse events (AEs) were similar between the groups receiving bempedoic acid and placebo with 86.3% of patients on bempedoic acid and 85.0% of patients on placebo reporting any AE that started or worsened after the first dose.
