Used primarily in the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder causing severely elevated LDL cholesterol levels. 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 and chylomicrons in the intestines. By blocking this process, it significantly reduces the production of LDL cholesterol and other atherogenic lipoproteins. Typically prescribed as an adjunct to a low-fat diet and other lipid-lowering therapies, including statins and LDL apheresis when needed. Due to its mechanism, it can cause gastrointestinal side effects and liver fat accumulation, so regular monitoring is required.