Daiichi Sankyo Europe GmbH (?Daiichi Sankyo?) and Esperion Therapeutics, Inc. jointly announced, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions for the label update of both bempedoic acid (marketed as NILEMDO®) and the bempedoic acid /ezetimibe fixed dose combination (FDC) (marketed as NUSTENDI®), recommending their approval as treatments to reduce low-density lipoprotein cholesterol (LDL-C) and cardiovascular risk. The existing label of bempedoic acid (NILEMDO®) provides authorisation for adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet: in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin, or alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated. The CHMP recommended adopting an update to this label, with which bempedoic acid is also indicated in adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors: in patients on a maximum tolerated dose of a statin with or without ezetimibe, or alone or in combination with ezetimibe in patients who are statin-intolerant, or for whom a statin is contraindicated. The existing label of bempedoic acid /ezetimibe FDC tablet (NUSTENDI®) provides authorisation of its use in adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet: in combination with a statin in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin in addition to ezetimibe, or alone in patients who are either statin-intolerant or for whom a statin is contraindicated, and are unable to reach LDL-C goals with ezetimibe alone, in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets with or without statin.

Additionally, the CHMP recommends an update of the bempedoic acid /ezetimibe label to amend its indication in adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors: in patients on a maximum tolerated dose of a statin and not adequately controlled with additional ezetimibe treatment or, in patients who are either statin-intolerant, or for whom a statin is contraindicated, and not adequately controlled with ezetimibe treatment or, in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets. The CHMP positive opinions are based on analysis from the Phase 3 CLEAR (Cholesterol Lowering via Bempedoic Acid, an ATP citrate lyase (ACL)-Inhibiting Regimen) Outcomes trial. The study enrolled a total of 13,970 patients aged 18-85 years old, and took place at 1,250 sites in 32 countries, including 485 sites across Europe.

Results from the Phase 3 CLEAR Outcomes trial demonstrated: a 13% reduction in the relative risk of major adverse cardiovascular events defined as a four-component composite of death from cardiovascular (CV) causes, non-fatal myocardial infarction, non-fatal stroke or coronary revascularisation (MACE-4). Results of the key secondary endpoints and subgroup analyses have been published. The CHMP is a scientific committee of the EMA that reviews medical product applications on their scientific and clinical merit.

The European Commission will review the CHMP opinions and is expected to deliver its final decision in the mid of the year 2024. NEXLETOL® (bempedoic acid) Tablet/NEXLIZET® (bempedoic acid and ezetimibe) Tablet is indicated as an adjunct to diet and statin therapy for the treatment of primary hyperlipidemia in adults with heterozygous familial hypercholesterolemia (HeFH) or atherosclerotic cardiovascular disease, who require additional lowering of LDL-C.