dm+d

38924811000001106

New Medicines

Nustendi (EU); Nexlizet (US)Hypercholesterolaemia, atherosclerotic cardiovascular disease (ASCVD) and/or heterozygous familial hypercholesterolemia (HeFH)

Information

Nustendi (EU); Nexlizet (US)
New formulation
Daiichi Sankyo
Esperion

Development and Regulatory status

Launched
Launched
Approved (Licensed)
December 2020
Dec 20Available in UK. 180mg/10mg blue oval f-c tab marked ESP and 818, 28=£55.44 [12].
Mar 20Approved in EU [11].
Feb 20Approved in the US, indicated 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), who require additional lowering of LDL-C. Launch is planned for Jul 2020 and will be sold at a wholesale acquisition cost of $10 per day [10].
Jan 20Recommended for EU approval by CHMP - the full indication is "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 (see sections 4.2, 4.3, and 4.4); 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” [20].
May 19The US FDA accepted the NDA for bemepedoic acid-ezetimibe and assigned the PDUFA goal date of Feb 26th 2020 [9].
Mar 19EU Marketing Authorisation Application validated by EMA [5].
Jan 19Daiichi Sankyo Europe will market oral bempedoic acid and bempedoic acid/ ezetimibe combination tablet in the European Economic Area, the U.K. and Switzerland [7].
Mar 18Esperion Therapeutics intends to submit a Marketing Authorization Application (MAA) for bempedoic acid/ezetimibe to the EMA by Q2 2019, and to file in the US in Q1 19 [3].

Category

First-in-class, non-statin targeted therapy that works in the liver to block cholesterol biosynthesis (bempedoic acid) plus a selective inhibitor of intestinal absorption of cholesterol and related phytosterols (ezetimibe).
Over half of all adults in England have raised cholesterol (>5mmol/L). Genetically inherited high cholesterol, or familial hypercholesterolemia, affects 1 in 250 or approximately 260,000 people in the UK [1].
Hypercholesterolaemia, atherosclerotic cardiovascular disease (ASCVD) and/or heterozygous familial hypercholesterolemia (HeFH)
Oral

Trial or other data

Aug 19Results from a 12-week PII study comparing a combination of bempedoic acid and ezetimibe to placebo and ezetimibe has published results showing that the combination lowers LDL-C by 40% compared to the control arm (p<0.001), and there was no worsening of glycaemic control. These two outcomes were important in this trial enrolling patients with hypercholesteralaemia and T2DM. A FDUFA date in February 2020 has been set [6].
Aug 18Initial results reported from 12-week PIII study (n=382). Patients allocated to fixed-dose bempedoic acid/ezetimibe combination achieved 35% reduction in LDL-C vs. 3% for placebo, 24% for ezetimibe, and 20% for bempedoic acid [4].
Oct 17Pivotal PIII trial to evaluate the efficacy and safety of bempedoic acid/ezetimibe (180/10 mg) fixed-dose combination vs. bempedoic acid, ezetimibe, and placebo alone in patients treated with maximally tolerated statin therapy starts (NCT03337308). Primary outcome is percent change in low-density lipoprotein cholesterol (LDL-C) at 12 weeks. The trial is designed to enrol approximately 350 patients with hypercholesterolaemia and ASCVD and/or HeFH, including high cardiovascular risk primary prevention patients, whose LDL-C is not adequately controlled despite receiving maximally tolerated lipid-modifying background therapy, in the US. Collection of primary outcome data is due to complete Jun 18 [2].

Evidence based evaluations