Unlike other therapies, lomitapide does not work by trying to clear the high levels of cholesterol. It works by reducing the production and release of cholesterol from the liver and by reducing the absorption of cholesterol from the intestines following a meal. Specifically, lomitapide is an oral, selective inhibitor of microsomal triglyceride transfer protein (MTP) a protein necessary for the proper assembly and secretion of cholesterol containing molecules in the liver and the intestines. Inhibition of MTP reduces the levels of cholesterol containing molecules including cholesterol, LDL-C and triglycerides in the blood.
The U.S. Food & Drug Administration (FDA) granted authorisation for lomitapide under the trade name ‘Juxtapid’ in December 2012. Juxtapid® is indicated as:
An adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-highdensity lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
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