Lomitapide
Lomitapide is a drug used as a lipid-lowering agent for the treatment of familial hypercholesterolemia, developed by Aegerion Pharmaceuticals.
It has been tested in clinical trials as single treatment and in combinations with atorvastatin, ezetimibe and fenofibrate.
The US Food and Drug Administration approved lomitapide on 21 December 2012, as an orphan drug to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia.
On 31 May 2013 the European Committee for Medicinal Products for Human Use adopted a positive opinion with a unanimous vote recommending a marketing authorization for lomitapide. On 31 July 2013 the European Commission approved lomitapide as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein apheresis in adult patients with HoFH.Mechanism of action
Lomitapide inhibits the microsomal triglyceride transfer protein which is necessary for very low-density lipoprotein assembly and secretion in the liver.
On 24 December 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."Side effects
In a Phase III study, lomitapide led to elevated aminotransferase levels and fat accumulation in the liver.
