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Review
. 2015 May;18:28-34.
doi: 10.1016/j.atherosclerosissup.2015.02.005.

Mipomersen and Lomitapide: Two New Drugs for the Treatment of Homozygous Familial Hypercholesterolemia

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Review

Mipomersen and Lomitapide: Two New Drugs for the Treatment of Homozygous Familial Hypercholesterolemia

Ioanna Gouni-Berthold et al. Atheroscler Suppl. .

Abstract

Familial hypercholesterolemia (FH) is a disease associated with very high plasma concentrations of low-density lipoprotein cholesterol (LDL-C) and premature cardiovascular disease. It is difficult in these high risk patients, exposed lifelong to very high LDL-C, to reach target LDL-C concentrations, which require >50% LDL-C reduction, even when on maximally tolerated statin therapy and on apheresis if available. Therefore, there is an unmet need for new therapeutic options for these patients. In 2013 two new drugs were approved for the treatment of homozygous FH, namely the apolipoprotein B synthesis inhibitor mipomersen and the microsomal transfer protein inhibitor lomitapide. Objective of this narrative review is to discuss the available evidence on the safety and efficacy profile of these new drugs.

Keywords: Apolipoprotein B synthesis inhibitor; Familial hypercholesterolemia; Homozygous form; Microsomal transfer protein (MTP) inhibitor.

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