Full Press Release Details
Positive CHMP Opinion for the Marketing Authorisation Application for Bempedoic Acid for the Treatment of Hypercholesterolemia
and Mixed Dyslipidemia
- Bempedoic Acid is an Oral, Once-Daily, Non-Statin Medicine that Lowers Bad Cholesterol with a First-in-Class Mechanism -
- Positive CHMP Opinion is Based on the Completed Pivotal Phase 3 LDL-Cholesterol Lowering Program -
- European Commission Decision on the Marketing Authorisation Application (MAA) Expected in April 2020; Daiichi Sankyo Europe to Lead EU Commercialization -
ANN ARBOR, Mich. January 31, 2020 (GLOBE
NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that the Committee for Medicinal Products for Human Use (CHMP) of
the European Medicines Agency (EMA) has adopted a positive opinion for the MAA for the bempedoic acid tablet, recommending approval
for the treatment of hypercholesterolemia and mixed dyslipidemia. The positive CHMP opinion was achieved with no Oral Explanation
as the Rapporteurs found there were no substantive issues that needed to be discussed with the CHMP at the time of the vote.
The benefits with the bempedoic acid
tablet are its ability to reduce levels of LDL-C, but also non-high-density lipoprotein cholesterol (non HDL-C), apolipoprotein
B (apo B), and total cholesterol (TC) in patients with hypercholesterolemia or mixed dyslipidemia when administered alone and in
combination with other lipid-modifying medicinal products. The
most common side effects are hyperuricemia, pain
in extremity, and anaemia.
The CHMP recommended granting the bempedoic
acid tablet marketing authorisation for the treatment of adults with primary hypercholesterolemia (heterozygous familial and non-familial)
or mixed dyslipidemia as an adjunct to diet:
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 opinion and is expected to adopt a final decision in April 2020. The decision will be applicable to all 27 European Union
member states plus the United Kingdom, Iceland, Norway and Liechtenstein.
"Today's positive recommendations from the CHMP
brings bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablets one step closer to helping the millions
of patients in the European Economic Area who have not achieved their LDL-C goals despite currently available medicines including
those who are statin intolerant," said Tim M. Mayleben, president and chief executive officer of Esperion. "We
look forward to continuing to work with the EMA to complete the procedure for these two MAAs by next quarter."
"This is the first Positive
Regulatory Agency acceptance for an ACL inhibitor and for our medicines," said Ashley Hall, chief development officer of
Esperion. "Today's decisions validate bempedoic acid and the bempedoic acid / ezetimibe combination tablets
for the treatment of hypercholesterolemia and mixed dyslipidemia. It also demonstrates the results from the remarkable efforts
of our Esperion Team to bring these new medicines to the community of physicians and their patients."
The positive CHMP opinion for the bempedoic acid tablet is supported by the global pivotal Phase 3 LDL-C Lowering Program conducted
in more than 4,000 patients. The bempedoic acid tablet provided up to 18 percent placebo corrected LDL-C lowering when used with
moderate- and high-intensity statins and 21 to 28 percent placebo corrected LDL-C lowering when used with low dose or no background
statin. Results from the Phase 3 development program have been published in The New England Journal of Medicine, The
Journal of the American Medical Association, The Journal of the American Heart Association, and Atherosclerosis.
Daiichi Sankyo Europe has licensed
exclusive commercialization rights to bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablets in the European
Economic Area and Switzerland from Esperion. Daiichi Sankyo's European cardiovascular commercial capabilities include more
than 1,000 professionals dedicated to the commercialization of cardiovascular medicines and includes synergies with an existing
portfolio of novel oral anticoagulant and antiplatelet products. Upon approval, Daiichi Sankyo Europe intends to make bempedoic
acid and the bempedoic acid / ezetimibe fixed dose combination tablets available to physicians and their patients in these geographies
that need treatment for hypercholesterolemia and mixed dyslipidemia.
Bempedoic acid and the bempedoic acid
/ ezetimibe fixed dose combination tablets are being developed globally as affordable, convenient, oral, once-daily, non-statin
medicines for the treatment of patients with elevated LDL-cholesterol (LDL-C) who are not at goal. Bempedoic acid and the bempedoic
acid 180 mg + ezetimibe 10 mg fixed dose combination tablets new drug applications (NDAs) are also under regulatory review by the
U.S. Food and Drug Administration (FDA) with PDUFA dates of February 21st and 26th 2020.
Bempedoic acid is our lead, oral, once-daily, non-statin, LDL-C
lowering therapeutic candidate, currently under regulatory review by the FDA and EMA. With a targeted mechanism of action, bempedoic
acid is a first-in-class, ATP Citrate Lyase (ACL) inhibitor that lowers LDL-C by reducing cholesterol biosynthesis and up-regulating
the LDL receptor. Bempedoic acid has been observed to reduce high sensitivity C-reactive protein (hsCRP), a key marker of inflammation
associated with cardiovascular disease. Completed Phase 3 studies conducted in more than 4,000 patients, with over 2,600 patients
treated with bempedoic acid, demonstrated up to 18 percent placebo corrected LDL-C lowering when used with moderate- and high-intensity
statins and 21 to 28 percent placebo corrected LDL-C lowering when used with low dose or no background statin. Phase 3 results
also show bempedoic acid reduced hsCRP 19 to 31 percent. Pooled phase 3 data highlighted that bempedoic acid reduced hemoglobin
A1c (HbA1c) by 0.19% versus placebo in patients with diabetes (n=1,134) at 12 weeks.
Bempedoic Acid / Ezetimibe Fixed
Dose Combination Tablet
Through the complementary mechanisms
of action of inhibition of cholesterol synthesis (bempedoic acid) and inhibition of cholesterol absorption (ezetimibe), the bempedoic
acid / ezetimibe fixed dose combination tablet is a non-statin, oral, once-daily, LDL-C lowering therapeutic candidate, currently
under review by the FDA and EMA. Inhibition of ATP Citrate Lyase (ACL) by bempedoic acid lowers LDL-C by reducing cholesterol biosynthesis
and up-regulating the LDL receptor. Inhibition of Niemann-Pick C1-Like 1 (NPC1L1) by ezetimibe results in reduced absorption of
cholesterol from the gastrointestinal tract, thereby reducing delivery of cholesterol to the liver. Phase 3 data demonstrated that
this combination resulted in a 29 percent placebo corrected LDL-C lowering when used with maximally tolerated statins, a 44 percent
LDL-C lowering when used with no background statin (post-hoc analysis), and a 34 percent reduction in high sensitivity C-reactive
CLEAR Cardiovascular Outcomes
The effect of bempedoic acid on cardiovascular
morbidity and mortality has not yet been determined. Esperion initiated a global cardiovascular outcomes trial (CVOT) to assess
the effects of bempedoic acid on the occurrence of major cardiovascular events in patients with, or at high risk for, cardiovascular
disease (CVD) who are only able to tolerate less than the lowest approved daily starting dose of a statin and considered "statin
averse." The CVOT - known as CLEAR Cardiovascular Outcomes Trial - is an event-driven, global, randomized, double-blind,
placebo-controlled study that completed enrollment in August 2019 of 14,032 patients with hypercholesterolemia and high CVD risk
at over 1,400 sites in 32 countries.
Esperion Therapeutics' Commitment
to Patients with Primary Hypercholesterolemia (heterozygous familial and non-familial) or Mixed Dyslipidemia
High levels of LDL-C can lead to a
build-up of fat and cholesterol in and on artery walls (known as atherosclerosis), potentially leading to cardiovascular events,
including heart attack or stroke. In the U.S., 96 million people, or more than 37 percent of the adult population, have elevated
LDL-C. There are approximately 18 million people in the U.S. with atherosclerotic cardiovascular disease (ASCVD) who live with
elevated levels of LDL-C despite taking maximally tolerated lipid-modifying therapy - including individuals considered statin
averse - leaving them at high risk for cardiovascular events1. In the United States, more than 50 percent of ASCVD
patients who are not able to reach their LDL-C goals with statins alone need less than a 40 percent reduction to reach their LDL-C
Esperion's mission as the Lipid Management
Company is to deliver oral, once-daily, non-statin, medicines that complement existing oral drugs to provide the additional LDL-C
lowering that these patients need.
Esperion Therapeutics
Through scientific and clinical excellence,
and a deep understanding of cholesterol biology, the experienced Lipid Management Team at Esperion is committed to developing new
LDL-C lowering medicines that will make a substantial impact on reducing global cardiovascular disease, the leading cause of death
Forward-Looking Statements
This press release contains forward-looking
statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding
the regulatory approval pathway for bempedoic acid tablet and the bempedoic acid / ezetimibe fixed dose combination tablet, the