Lomitapide mesylateProduct ingredient for Lomitapide

Lomitapide, marketed under the brand names Juxtapid (USA) and Lojuxta (EU), is a first-in-class, small-molecule inhibitor of microsomal triglyceride transfer protein (MTP).^1,5 It was developed to address the significant unmet medical need in patients with homozygous familial hypercholesterolemia (HoFH)—an ultra-rare and life-threatening genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) and premature atherosclerotic cardiovascular disease.^1,2. Unlike traditional statin therapy, which relies on the upregulation of LDL receptors to clear cholesterol, Lomitapide acts independently of the LDL receptor by preventing the assembly and secretion of apo B-containing lipoproteins in the liver and intestines.^2,5 This makes it a critical therapeutic option for "receptor-negative" patients who have little to no functional LDL receptor activity.⁵ Lomitapide was first approved by the FDA in December 2012 and the EMA in July 2013 as an adjunct to a low-fat diet and other lipid-lowering treatments.^1,3