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Lomitapide: A Review of its Use in Adults with Homozygous Familial Hypercholesterolemia

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Abstract

Lomitapide (JuxtapidTM), an orally administered inhibitor of the microsomal triglyceride transfer protein, inhibits the synthesis of chylomicrons and very low-density lipoprotein, thereby reducing plasma levels of low-density lipoprotein cholesterol (LDL-C). Lomitapide is used to lower lipid levels in adults with homozygous familial hypercholesterolemia, a rare, potentially life-threatening genetic disease that is commonly caused by mutations in the LDL receptor gene or other genes that affect the function of the LDL receptor. In a multinational single-arm, open-label, 78-week, phase III trial, lomitapide reduced mean plasma LDL-C levels by 50 % from baseline in 23 evaluable adults with homozygous familial hypercholesterolemia over a 26 week treatment period. Reductions from baseline in LDL-C levels were sustained for up to 78 weeks with continued lomitapide treatment. In this study, the initial dosage of lomitapide was 5 mg once daily for two weeks, with upward titration thereafter to 10, 20, 40, and 60 mg at weeks 2, 6, 10, and 14, respectively, or until an individually assessed maximum dosage was achieved. Prior to the start of treatment with lomitapide, other lipid-lowering therapy (including LDL apheresis) was stabilized over a 6-week period, and then continued throughout the lomitapide treatment phase. Lomitapide was generally well tolerated; the most common adverse events in the phase III trial were gastrointestinal events.

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Disclosure

The preparation of this review was not supported by any external funding. During the peer review process, the manufacturer of the agent under review was offered an opportunity to comment on this article; comments provided are only related to accuracy and completeness of the information. Changes resulting from comments received were made by the authors on the basis of scientific and editorial merit.

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  1. Adis, 41 Centorian Drive, Private Bag 65901 Mairangi Bay, North Shore, 0754, Auckland, New Zealand

    Caroline M. Perry

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Correspondence to Caroline M. Perry.

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Perry, C.M. Lomitapide: A Review of its Use in Adults with Homozygous Familial Hypercholesterolemia. Am J Cardiovasc Drugs 13, 285–296 (2013). https://doi.org/10.1007/s40256-013-0030-7

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