Abstract
Familial hypercholesterolemia (FH) is a common genetic condition characterized by elevated plasma levels of low-density lipoprotein cholesterol (LDL-C), premature atherosclerotic cardiovascular disease, and considerable unmet medical need with conventional LDL-C-lowering therapies. Between 2012 and 2015, the US Food and Drug Administration approved four novel LDL-C-lowering agents for use in patients with FH based on the pronounced LDL-C-lowering efficacy of these medicines. We review the four novel approved agents, as well as promising LDL-C-lowering agents in clinical development, with a focus on their mechanism of action, efficacy in FH cohorts, and safety.
Keywords:
LDL; lomitapide; low-density lipoprotein; mipomersen; proprotein convertase subtilisin/kexin 9.
MeSH terms
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Angiopoietin-like Proteins / antagonists & inhibitors
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Antibodies, Monoclonal / therapeutic use
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Antibodies, Monoclonal, Humanized
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Anticholesteremic Agents / therapeutic use*
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Benzimidazoles / therapeutic use
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Caproates / therapeutic use
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Cholesterol Ester Transfer Proteins / antagonists & inhibitors
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Dicarboxylic Acids / therapeutic use
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Fatty Acids / therapeutic use
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Genetic Therapy
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Humans
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Hyperlipoproteinemia Type II / therapy*
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Oligonucleotides / therapeutic use
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RNA, Small Interfering / therapeutic use
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Receptors, LDL / genetics
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Treatment Outcome
Substances
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ALN-PCS
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Angiopoietin-like Proteins
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Antibodies, Monoclonal
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Antibodies, Monoclonal, Humanized
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Anticholesteremic Agents
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BMS201038
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Benzimidazoles
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Caproates
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Cholesterol Ester Transfer Proteins
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Dicarboxylic Acids
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Fatty Acids
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LDLR protein, human
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Oligonucleotides
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RNA, Small Interfering
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Receptors, LDL
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8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid
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mipomersen
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gemcabene
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evolocumab
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alirocumab