What is the role of bempedoic acid in the treatment of hypercholesterolemia (high cholesterol)?

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Bempedoic Acid in Hypercholesterolemia Management

Bempedoic acid is an effective adjunct therapy for patients with hypercholesterolemia who require additional LDL-C lowering beyond maximally tolerated statin therapy, particularly valuable for statin-intolerant patients due to its liver-selective activation that avoids muscle-related side effects. 1, 2

Mechanism of Action

Bempedoic acid works through a novel mechanism:

  • Inhibits ATP-citrate lyase (ACL), an enzyme upstream of HMG-CoA reductase in the cholesterol biosynthesis pathway 3
  • Functions as a prodrug that requires activation by very long-chain acyl-CoA synthetase 1 (ACSVL1), which is primarily expressed in the liver but not in muscle tissue 2, 3
  • This liver-selective activation provides a key advantage for patients with statin-associated muscle symptoms 1, 2
  • Reduces acetyl-CoA availability for cholesterol synthesis 2
  • Upregulates LDL receptors, improving LDL clearance from the bloodstream 3

Efficacy

Bempedoic acid demonstrates significant lipid-lowering effects:

  • Reduces LDL-C by approximately 15-25% when added to maximally tolerated statin therapy 2
  • Monotherapy reduces LDL-C by approximately 24.5% in statin-intolerant patients 1
  • When combined with ezetimibe, provides up to 38% additional LDL-C reduction 1, 2
  • Also significantly lowers:
    • Non-HDL cholesterol
    • Total cholesterol
    • Apolipoprotein B
    • High-sensitivity C-reactive protein (hs-CRP) by approximately 24-28% 2, 4

Cardiovascular Outcomes

The CLEAR Outcomes trial demonstrated significant cardiovascular benefits:

  • 13% reduction in composite major adverse cardiovascular events (MACE) compared to placebo 2, 4
  • 24% reduction in myocardial infarction risk 4
  • 31% reduction in unstable angina 4
  • Greater benefit in primary prevention (30% reduction) compared to secondary prevention 2
  • 17% reduction in MACE in patients with diabetes 2

Clinical Applications

Bempedoic acid is FDA-approved for:

  • Adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD) who require additional LDL-C lowering 1, 2
  • Use as an adjunct to diet and maximally tolerated statin therapy 1
  • Available as monotherapy (180 mg daily) or in fixed-dose combination with ezetimibe 10 mg 1, 2

Patient Selection

Bempedoic acid is particularly valuable for:

  • Patients with statin intolerance due to muscle-related side effects 1, 2
  • Patients who have not achieved ≥50% LDL-C reduction and LDL-C <70 mg/dL on maximally tolerated statin therapy 2
  • Patients with established ASCVD or HeFH requiring additional LDL-C lowering 1, 2
  • Patients with adherence issues who may benefit from the combination preparation with ezetimibe 1

Safety Profile

Bempedoic acid has a generally favorable safety profile with some notable considerations:

  • Overall safety comparable to placebo in most aspects 4
  • Increased risk of gout (1.2-1.5% vs. 0.3-0.4% with placebo) due to elevated uric acid levels 1, 4
  • Slight increases in:
    • Tendon rupture (0.5% vs. 0%) 1
    • Benign prostatic hyperplasia (1.3% vs. 0.1%) 1
    • Atrial fibrillation (1.7% vs. 1.1%) 1
    • Elevated creatine kinase levels (1.0% vs. 0.6%) 1
  • Higher incidence of adverse events leading to discontinuation (10.9% vs. 7.1% with placebo) 5

Pharmacokinetics

Key pharmacokinetic properties include:

  • Oral administration with median time to maximum concentration of 3.5 hours 3
  • Food does not affect bioavailability 3
  • Mean half-life of 21 ± 11 hours at steady-state 3
  • Steady-state achieved after 7 days 3
  • Primary elimination through glucuronide metabolism 3
  • No dose adjustment needed with food 3

Clinical Considerations

Important considerations when prescribing bempedoic acid:

  • Monitor for elevated uric acid levels and symptoms of gout 1, 2
  • Watch for potential tendon disorders 2
  • Consider cost implications as it is available as a branded product 1
  • Patient assistance programs and discount copay cards are available for eligible patients 1
  • May be considered after ezetimibe or PCSK9 inhibitors in the treatment algorithm 2

Bempedoic acid represents an important addition to the lipid-lowering armamentarium, particularly for patients who cannot tolerate statins or require additional LDL-C reduction despite maximally tolerated statin therapy.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Guideline

Lipid Management with Bempedoic Acid Therapy

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Research

Safety and Efficacy of Bempedoic Acid to Reduce LDL Cholesterol.

The New England journal of medicine, 2019

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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