Introduction
Aegerion Pharmaceuticals is developing lomitapide, a small- molecule,microsomaltriglyceridetransferprotein(MTP)inhibitor, for the treatment of hypercholesterolemia, both familial and primary. Microsomal triglyceride transfer protein inhibitors have a dual mechanism to reduce both LDL and triglyceride levels. The compound is being developed as an oral, once-a-day treatment. Lomitapide will be targeted at patients resistant to statins either due to abnormalities in liver function or to dis- continuation because of muscle pain. An oral formulation of lomitapideis in phase IIIdevelopment forhomozygous familial hypercholesterolemia (HoFH; hyperlipoproteinemia type IIa) in the US, Canada, Italy, and South Africa. Aegerion intends to develop lomitapide also for the treat- ment of familial chylomicronemia (FC; hyperlipoproteinemia type I). Lomitapide is expected to reduce triglyceride levels in patients with FC.