Mipomersen and lomitapide: Two new drugs for the treatment of homozygous familial hypercholesterolemia.
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.
Center of Endocrinology, Diabetes and Preventive Medicine (ZEDP), University of Cologne, Kerpener Str. 62, 50937 Cologne, Germany. Electronic address: ioanna.berthold@uni-koeln.de.
Ioanna
I
Gouni-Berthold
Department of Internal Medicine and Geriatrics, Bielefeld Evangelical Hospital (EvKB), Schildescher Str. 99, 33611 Bielefeld, Germany.
Heiner K
HK
Berthold
D000818:Animals; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D053299:Apolipoprotein B-100 / Q000096:biosynthesis / Q000235:genetics; D001562:Benzimidazoles / Q000009:adverse effects / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood; D002352:Carrier Proteins / Q000037:antagonists & inhibitors / Q000378:metabolism; D008078:Cholesterol, LDL / Q000097:blood*; D020022:Genetic Predisposition to Disease; D006720:Homozygote; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics; D009154:Mutation; D009841:Oligonucleotides / Q000009:adverse effects / Q000627:therapeutic use*; D010641:Phenotype; D011973:Receptors, LDL / Q000235:genetics*; D016896:Treatment Outcome
C569471:APOB protein, human; D000924:Anticholesteremic Agents; D053299:Apolipoprotein B-100; C473731:BMS201038; D001562:Benzimidazoles; D015415:Biomarkers; D002352:Carrier Proteins; D008078:Cholesterol, LDL; C573412:LDLR protein, human; D009841:Oligonucleotides; D011973:Receptors, LDL; C106092:microsomal triglyceride transfer protein; C524142:mipomersen
Apolipoprotein B synthesis inhibitor; Familial hypercholesterolemia; Homozygous form; Microsomal transfer protein (MTP) inhibitor