CHMP issues positive opinions for both bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablet as treatments for primary hypercholesterolaemia or mixed dyslipidaemia

• Bempedoic acid is an oral, once-daily ATP citrate lyase inhibitor that reduces cholesterol synthesis in the liver. This unique mechanism of action is synergistic to statins and other lipid-lowering therapies[i]
• Positive CHMP opinions are based on a global clinical trial programme demonstrating consistent LDL-C lowering across a range of patient populations[ii]^,[iii]^{,[iv],[v],-[vi]}
• European Commission decisions on Marketing Authorisation Application expected in the first half of 2020

MUNICH, Germany, (January 31, 2020) – Daiichi Sankyo Europe GmbH (hereafter, ‘Daiichi Sankyo’) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions for the marketing authorisations of both bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination (FDC) tablet, recommending their approval as treatments to reduce low-density lipoprotein cholesterol (LDL-C).

Bempedoic acid is being developed as a first-in-class, affordable, convenient, once-daily, oral therapy for people with primary hypercholesterolaemia or mixed dyslipidaemia.

The CHMP recommended granting bempedoic acid marketing 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 granting the bempedoic acid / ezetimibe FDC tablet marketing authorisation for 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,

·                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 a statin.

“Cardiovascular disease is the leading cause of death in Europe, killing over four million people every year, and increased LDL-C levels are one of the most important risk factors. However, as many as 80% of people on statins are not reaching their LDL-C targets.[vii] This is challenging for patients and their healthcare professionals,” said Benoit Creveau, Head of Cardiovascular Marketing at Daiichi Sankyo Europe. “The clinical development programme for bempedoic acid demonstrated consistent lowering of LDL-C in a broad range of patients, including those already receiving maximally tolerated oral lipid-lowering therapies. The CHMP opinion is an acknowledgement of these results and brings us one step closer to our commitment of helping patients in the European Union who have not yet achieved their LDL-C goals.”

The CHMP positive opinions are supported by a global pivotal Phase III clinical trial programme conducted in more than 4,000 patients. Bempedoic acid provided up to 18% placebo-corrected LDL-C lowering when used with moderate- and high-intensity statins and 21-28% placebo-corrected LDL-C lowering when used with low-dose or no background statin.3^,4,5 The bempedoic acid / ezetimibe FDC tablet provided a 38% placebo-corrected LDL-C lowering when used with maximally-tolerated statin therapy, which may mean no statin at all.6

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 first half of 2020. Bempedoic acid and the bempedoic acid / ezetimibe FDC tablet are also currently under regulatory review by the U.S. Food and Drug Administration (FDA) with PDUFA dates of February 21^st and 26^th 2020.

[i] Nickolic D, et al. ETC-1002: A Future Option for Lipid Disorders? Atherosclerosis 2014 Dec;237(2):705-10.

[ii] Goldberg AC, et al. Effect of Bempedoic Acid vs Placebo Added to Maximally Tolerated Statins on Low-Density Lipoprotein Cholesterol in Patients at High Risk for Cardiovascular Disease. The CLEAR Wisdom Randomized Clinical Trial. JAMA. 2019;322(18):1780-1788.e

[iii] Ray KK, et al. Safety and Efficacy of Bempedoic Acid to Reduce LDL Cholesterol. N Engl J Med. 2019;380:1022–32

[iv] Laufs U, et al. Efficacy and Safety of Bempedoic Acid in Patients with Hypercholesterolemia and Statin Intolerance. J Am Heart Assoc. 2019;8(7):e011662.

[v] Ballantyne CM, et al. Efficacy and safety of bempedoic acid added to ezetimibe in statin-intolerant patients with hypercholesterolemia: A randomized, placebo-controlled study. Atherosclerosis.2018;277:195-203.

[vi] Ballantyne, CM, et al. Bempedoic acid plus ezetimibe fixed-dose combination in patients with hypercholesterolemia and high CVD risk treated with maximally tolerated statin therapy. Eur J Prev Cardiol. 2019; doi 10.1177/2047487319864671.

[vii] Fox KM, Tai M-H, Kostev K et al. Treatment patterns and low-density lipoprotein cholesterol (LDL-C) goal attainment among patients receiving high- or moderate-intensity statins. Clin Res Cardiol. 2018; 107: 380 –388.