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A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
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Bempedoic Acid for Heterozygous Familial Hypercholesterolemia: From Bench to Bedside

Anandita Agarwala1, Renato Quispe2, Anne C Goldberg3

  • 1Division of Cardiology, Baylor Scott and White Health, Heart Hospital Baylor Plano, Plano, TX, USA.

Drug Design, Development and Therapy
|May 19, 2021
PubMed

Insights

Bempedoic acid effectively lowers LDL-C in patients with heterozygous familial hypercholesterolemia (HeFH) and those intolerant to statins. This cholesterol biosynthesis inhibitor offers a new option for atherosclerotic cardiovascular disease (ASCVD) prevention.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Biochemistry

Background:

  • Bempedoic acid is a novel oral cholesterol biosynthesis inhibitor.
  • It is approved for atherosclerotic cardiovascular disease (ASCVD) and heterozygous familial hypercholesterolemia (HeFH).

Purpose of the Study:

  • To review the profile of bempedoic acid, including its design and development.
  • To discuss its therapeutic role in managing low-density lipoprotein cholesterol (LDL-C) for ASCVD prevention.

Main Methods:

  • Review of pooled data from Phase III clinical trials (CLEAR Harmony and CLEAR Wisdom).
  • Discussion of ongoing cardiovascular outcomes trial (CLEAR Outcomes).

Main Results:

  • Phase III trials demonstrated bempedoic acid's safety and efficacy in lowering LDL-C.
  • It serves as an adjunct or alternative to existing lipid-lowering therapies for HeFH patients.

Conclusions:

  • Bempedoic acid is a valuable option for patients needing additional LDL-C lowering, especially those with HeFH or statin intolerance.
  • Further insights into its role in non-statin lipid-lowering therapy are expected from CLEAR Outcomes.

Keywords:
atherosclerotic cardiovascular diseasebempedoic acidfamilial heterozygous hypercholesterolemialipidslow-density lipoprotein cholesterolprevention

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