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Lomitapide + niacin is a combination of two lipid-lowering drugs, used primarily as adjunct therapies in patients with severe dyslipidemia such as homozygous familial hypercholesterolemia (HoFH). Lomitapide is a small molecule inhibitor of microsomal triglyceride transfer protein (MTP), suppressing the assembly and secretion of very-low-density lipoproteins (VLDL) and chylomicrons, ultimately reducing circulating levels of low-density lipoprotein cholesterol (LDL-C), total cholesterol, apolipoprotein B, and non-HDL cholesterol. Niacin (nicotinic acid) is a water-soluble B vitamin that, in pharmacological doses, reduces LDL-C, triglycerides, and lipoprotein(a), and notably increases high-density lipoprotein cholesterol (HDL-C) by inhibiting hepatic diacylglycerol acyltransferase-2, decreasing hepatic VLDL synthesis, and reducing catabolism of apoA-I. The combination of these agents targets multiple aspects of lipid metabolism and is considered in patients requiring intensive lipid modification not attainable with monotherapy or standard agents. There is no uniquely approved or branded formulation containing both lomitapide and niacin; the combination is considered an off-label strategy rather than an established fixed-dose product[1][5][6][7].
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