• Dave L Dixon, Evan M Sisson, Michael Butler, Ashley Higbea, Brendan Muoio, and Brandy Turner.
• Dave L. Dixon, PharmD, BCPS, CDE, CLS Assistant Professor, School of Pharmacy, Department of Pharmacotherapy & Outcomes Science, Virginia Commonwealth University, Richmond. Evan M. Sisson, PharmD, MHA, CDE Associate Professor, School of Pharmacy, Department of Pharmacotherapy & Outcomes Science, Virginia Commonwealth University. Michael Butler, PharmD Student, School of Pharmacy, Virginia Commonwealth University, Richmond. Ashley Higbea, PharmD Student, School of Pharmacy, Virginia Commonwealth University, Richmond. Brendan Muoio, PharmD Student, Virginia Commonwealth University School of Pharmacy, Richmond. Brandy Turner, PharmD Student, School of Pharmacy, Virginia Commonwealth University, Richmond.
• J Cardiovasc Nurs. 2014 Sep 1; 29 (5): E7-E12.

BackgroundFamilial hypercholesterolemia (FH) is an autosomal dominant disorder caused primarily by mutations in the low-density lipoprotein receptor gene. Familial hypercholesterolemia is characterized by exceedingly high levels of low-density lipoprotein cholesterol (LDL-C) and subsequent premature coronary heart disease. Homozygous FH (HoFH) is less prevalent, but more severe, than heterozygous FH. Current treatment options include dietary therapy, lipid-lowering agents (eg, statins), and/or LDL-C apheresis.PurposeDespite the available treatment options, patients with FH rarely attain treatment goals. This review will focus on 2 novel agents, lomitapide and mipomersen, with recently approved US Food and Drug Administration (FDA) labeling for use in patients with HoFH.ConclusionsLomitapide and mipomersen are 2 agents with novel mechanisms of action and the ability to significantly lower LDL-C, apolipoprotein B, and non-high-density lipoprotein cholesterol levels. A black box warning exists for lomitapide and mipomersen regarding the risk for transaminase elevations and hepatic steatosis. Furthermore, these agents are currently restricted for use only in patients with HoFH and have been required by the FDA to participate in a Risk Evaluation and Mitigation Strategy.Clinical ImplicationsThese new agents offer additional treatment options for clinicians managing patients with HoFH, but it remains uncertain whether lomitapide and mipomersen will gain FDA approval for use in patients with heterozygous FH or in the general population. Cost and concern for the risk for hepatotoxicity will remain limiting factors to these agents being more widely used.

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