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.
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