Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder of lipid metabolism. 1,2 Clinical traits of HoFH comprise of elevated plasma levels of low-density lipoprotein cholesterol (LDL-C) from birth, cutaneous xanthomas, and accelerated atherosclerotic cardiovascular disease. 1,3 HoFH often leads to an increased risk of premature cardiovascular disease and complications before the age of 20 years when left untreated.
Lomitapide is a microsomal triglyceride transfer protein inhibitor that is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). It has been approved by the US Food and Drug…
