Lomitapide

Lomitapide (INN, marketed as Juxtapid) is an investigational drug for the treatment of familial hypercholesterolemia, developed by Aegerion Pharmaceuticals. It has been tested in several Phase II clinical trials as single treatment and in combinations with atorvastatin, ezetimibe and fenofibrate.

The US Food and Drug Administration approved lomitapide on December 21, 2012 as an orphan drug to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH).

Mechanism of action
Lomitapide inhibits the microsomal triglyceride transfer protein (MTP or MTTP) which is necessary for very low-density lipoprotein (VLDL) assembly and secretion in the liver.

On December 24, 2012, drug manufacturer Aegerion announced they had been approved by the FDA to as "an adjunct to a low-fat diet and other lipid-lowering treatments...in patients with homozygous familial hypercholesterolemia (HoFH)."

Side effects
In a Phase II study, lomitapide lead to elevated aminotransferase levels and fat accumulation in the liver.