Lomitapide is primarily used as a therapeutic agent for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) in the blood. It works by inhibiting the microsomal triglyceride transfer protein (MTP), which is essential for the assembly and secretion of very low-density lipoproteins (VLDL) in the liver. By blocking this process, lomitapide significantly reduces the production of LDL-C, helping to lower cholesterol levels in patients with HoFH. It is typically prescribed when other lipid-lowering therapies, such as statins or LDL apheresis, are insufficient. Due to its mechanism of action, lomitapide may cause gastrointestinal side effects and liver fat accumulation, requiring careful monitoring and dietary management during treatment. It is often used in combination with a low-fat diet and other lipid-lowering therapies to achieve optimal results.