The approval of bempedoic acid (Nexletol) marks the first oral, non-statin therapy approved by the FDA as a lipid-lowering agent in nearly 20 years.
Bempedoic acid (Nexletol)—a once-daily, oral non-statin treatment—has received approval from the US Food and Drug Administration as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD), according to labeling information from the FDA.
Backed by clinical trial data indicating bempedoic acid can reduce LDL-C by 18% when used as an adjunct to maximally-tolerated statin therapy and by 28% when used in statin averse patients, the first-in-class, ACL inhibitor will be a welcome and eagerly-anticipated addition to treatment armamentariums for many clinicians.
“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,” said Tim Mayleben, president and chief executive officer of Esperion. “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 approval of bempedoic acid is for a once-daily 180 mg dosage that the indication purports can be taken with or without food. The labeling information warns patients and clinicians that the effects of bempedoic acid on cardiovascular morbidity and mortality has not been evaluated.
Additionally, patients and clinicians should be aware bempedoic acid was associated with possible increase in hyperuricemia and tendon rupture. Labeling information also recommends avoiding concomitant use with simvastatin greater than 20 mg and pravastatin greater than 40 mg.
Using data from 4 trials examining use of the treatment for lowering elevated LDL-C, Esperion submitted a New Drug Application (NDA) for bempedoic acid in February 2019. From these studies, the FDA had information on more than 4000 patients with ASCVD or at high risk of ASCVD with HeFH—including more than 2600 patients receiving bempedoic acid.
Additionally, an analysis of pooled data of 4 phase 3 CLEAR trials presented at AHA 2019 also gives credence to bempedoic acid’s LDL-C lowering ability in patients with ASCVD or with HeFH and at high risk of ASCVD. The results of this analysis also indicated bemepdoic acid could also help improve on-HDL-C, total cholesterol, apolipoprotein B, and hsCRP from 12 weeks of treatment.
Esperion expects bempedoic acid to be available by March 30. Esperion noted an NDA for bempedoic acid/ezetimibe was filed along with the bempedoic acid NDA in 2019—this application has a PDUFA date of February 26, 2020.