Lomitapide: a review of its use in adults with homozygous familial hypercholesterolemia

Am J Cardiovasc Drugs. 2013 Aug;13(4):285-96. doi: 10.1007/s40256-013-0030-7.

Abstract

Lomitapide (Juxtapid(TM)), 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.

Publication types

  • Clinical Trial, Phase II
  • Clinical Trial, Phase III

MeSH terms

  • Anticholesteremic Agents / adverse effects
  • Anticholesteremic Agents / pharmacokinetics
  • Anticholesteremic Agents / therapeutic use*
  • Benzimidazoles / adverse effects
  • Benzimidazoles / pharmacokinetics
  • Benzimidazoles / therapeutic use*
  • Cholesterol, LDL / blood
  • Homozygote
  • Humans
  • Hyperlipoproteinemia Type II / blood
  • Hyperlipoproteinemia Type II / drug therapy*

Substances

  • Anticholesteremic Agents
  • BMS201038
  • Benzimidazoles
  • Cholesterol, LDL