New Lipid-Lowering Drug Earns FDA Approval
The US Food and Drug Administration has approved bempedoic acid (Nexletol, Esperion) for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD) who require additional low-density lipoprotein-cholesterol lowering.
The oral adenosine triphosphate-citrate lyase (ACL) inhibitor is indicated as an adjunct to diet and maximally tolerated statin therapy in these patients, and it is approved at the 180 mg once daily dose, the agency announced today.
The safety and efficacy of bempedoic acid were demonstrated over 52 weeks in two multicenter randomized clinical trials involving 3009 adults with HeFH or established ASCVD on maximally tolerated statin therapy.
The difference between bempedoic acid and placebo for the primary outcome of change in LDL-cholesterol from baseline to week 12 was –18% in the first trial (95% confidence interval [CI], –20% to –16%; P < .001) and –17% in the second trial (95% CI, –21% to –14%; P < .001).
The label notes that the effect on cardiovascular morbidity and mortality has not been determined.
The label also includes warnings stating that bempedoic acid may increase blood uric acid levels and is associated with an increased risk of tendon rupture or injury.
In clinical trials, 26% of bempedoic acid-treated patients with normal baseline uric acid values vs 9.5% of placebo-treated patients experienced hyperuricemia one or more times, and 3.5% of patients experienced clinically significant hyperuricemia reported as an adverse reaction vs 1.1% with placebo, according to the label.
Gout was reported in 1.5% of patients treated with bempedoic acid and 0.4% of those treated with placebo.
In clinical trials, the risk of tendon rupture was 0.5% with bempedoic acid and 0% with placebo. Tendon rupture involved the rotator cuff, biceps tendon, or Achilles tendon, and occurred within weeks to months of starting the drug.
Rupture may “occur more frequently in patients over 60 years of age, in those taking corticosteroid or fluoroquinolone drugs, in patients with renal failure, and in patients with previous tendon disorders,” the label states.
The label also advises patients to avoid concomitant use of bempedoic acid with simvastatin greater than 20 mg or pravastatin greater than 40 mg because it causes an increase in simvastatin and pravastatin concentrations and may increase the risk of related myopathy.
A decision is expected shortly on a new drug application submitted by Esperion for an LDL-cholesterol lowering indication for bempedoic acid 180 mg/ezetimibe 10 mg combination tablet.
Full prescribing information is available online.