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Bempedoic Acid – Mechanism, Uses & Side Effects

Bempedoic acid is a lipid-lowering medication used to reduce elevated LDL cholesterol levels, either as an add-on to statins or as an alternative for statin-intolerant patients.

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Things worth knowing about "Bempedoic acid"

Bempedoic acid is a lipid-lowering medication used to reduce elevated LDL cholesterol levels, either as an add-on to statins or as an alternative for statin-intolerant patients.

What is Bempedoic Acid?

Bempedoic acid is a lipid-lowering drug used to treat elevated levels of LDL cholesterol (low-density lipoprotein). It belongs to the class of ATP-citrate lyase (ACL) inhibitors and has been approved by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). It is marketed under the brand name Nexletol (as a monotherapy) and in combination with ezetimibe as Nexlizet (EU: Nilemdo and Nustendi). Bempedoic acid is intended for patients who do not achieve their LDL target values despite statin therapy or who cannot tolerate statins.

Mechanism of Action

Bempedoic acid works by inhibiting the enzyme ATP-citrate lyase (ACL), which catalyzes an early step in cholesterol biosynthesis in the liver. This step occurs upstream of the target of statins (HMG-CoA reductase), providing a complementary mechanism of action.

A key advantage is that bempedoic acid is a prodrug: it is only converted into its active form in the liver, meaning it acts predominantly at the hepatic level. In muscle tissue, it remains largely inactive, which significantly reduces the risk of muscle pain (myopathy) compared to statins.

Indications and Usage

Bempedoic acid is indicated for adults with:

  • Primary hypercholesterolemia (heterozygous familial or non-familial hypercholesterolemia)
  • Mixed dyslipidemia
  • Patients who are statin-intolerant
  • Patients who do not reach LDL target levels despite maximum tolerated statin therapy

The recommended dose is 180 mg once daily as an oral tablet, taken with or without food. Bempedoic acid can be used alone or in combination with other lipid-lowering agents such as statins or ezetimibe.

Clinical Efficacy

In clinical trials, particularly the landmark CLEAR Outcomes study (2023), bempedoic acid demonstrated significant benefits:

  • Reduction of LDL cholesterol by approximately 15–25%
  • Reduction of high-sensitivity C-reactive protein (hsCRP), a marker of systemic inflammation
  • Significant reduction in major cardiovascular events (heart attack, stroke) in high-risk patients who could not take statins

Side Effects

Bempedoic acid is generally well tolerated. The most common side effects include:

  • Hyperuricemia (elevated uric acid levels in the blood) and an associated increased risk of gout
  • Elevated liver enzymes (uncommon)
  • Muscle and tendon complaints, including an increased risk of tendon rupture
  • Gastrointestinal symptoms (e.g., abdominal pain, diarrhea)
  • Increase in serum creatinine levels

Due to the risk of tendon rupture, bempedoic acid should be used with caution in patients who are also taking fluoroquinolone antibiotics.

Contraindications and Interactions

Bempedoic acid should not be used in:

  • Patients with known hypersensitivity to the active substance
  • Pregnancy and breastfeeding (due to lack of safety data)

Important drug interactions include:

  • Simvastatin and Pravastatin: Bempedoic acid may increase plasma levels of these statins; dose adjustment may be required
  • Ciclosporin: concomitant use is contraindicated

References

  1. Ray KK et al. - Bempedoic Acid and Cardiovascular Outcomes in Statin-Intolerant Patients (CLEAR Outcomes). New England Journal of Medicine, 2023. DOI: 10.1056/NEJMoa2215024
  2. European Medicines Agency (EMA) - Summary of Product Characteristics: Nilemdo (bempedoic acid), EMA/CHMP Assessment Report, 2020. Available at: www.ema.europa.eu
  3. Ballantyne CM et al. - Bempedoic Acid plus Ezetimibe Fixed-Dose Combination in Patients with Hypercholesterolemia and High CVD Risk. European Journal of Preventive Cardiology, 2020; 27(6): 593–603.

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