title: Lomitapide.
abstract: Aegerion Pharmaceuticals is developing lomitapide, a small-molecule, microsomal triglyceride transfer protein (MTP) inhibitor, for the treatment of both familial and primary hypercholesterolemia. Oral, once-daily lomitapide will be targeted at patients resistant to HMG-CoA reductase inhibitors (statins) either due to abnormalities in liver function or to discontinuation because of muscle pain. An oral formulation of lomitapide is in phase III development for homozygous familial hypercholesterolemia (hyperlipoproteinemia type IIa) in the US, Canada, Italy, and South Africa. This review discusses the key development milestones and therapeutic trials of this drug.
mesh_terms: D000818:Animals; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D001562:Benzimidazoles / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D002352:Carrier Proteins / Q000037:antagonists & inhibitors*; D015507:Drugs, Investigational / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D006937:Hypercholesterolemia / Q000188:drug therapy*; D008072:Hyperlipoproteinemia Type I / Q000188:drug therapy; D006938:Hyperlipoproteinemia Type II / Q000188:drug therapy; D015228:Hypertriglyceridemia / Q000188:drug therapy; D000960:Hypolipidemic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: D000924:Anticholesteremic Agents; C473731:BMS201038; D001562:Benzimidazoles; D002352:Carrier Proteins; D015507:Drugs, Investigational; D000960:Hypolipidemic Agents; C106092:microsomal triglyceride transfer protein