Lomitapide mesylate

Lomitapide mesylate is a lipid-lowering agent used for the treatment of homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder characterized by high cholesterol levels. It is marketed under the brand name Juxtapid in the United States and Lojuxta in Europe.

Mechanism of Action

Lomitapide mesylate works by inhibiting the microsomal triglyceride transfer protein (MTP), which is found in the liver and intestines. MTP is responsible for the assembly of apolipoprotein B (apoB), a protein that is crucial for the production of low-density lipoprotein (LDL), often referred to as "bad cholesterol". By inhibiting MTP, lomitapide mesylate reduces the production of LDL and other apoB-containing lipoproteins, thereby lowering the levels of these substances in the blood.

Clinical Use

Lomitapide mesylate is used in conjunction with a low-fat diet and other lipid-lowering treatments in patients with HoFH. It is typically prescribed to patients who have not responded adequately to other treatments. The drug is available in capsule form and is taken orally once a day.

Side Effects

The most common side effects of lomitapide mesylate include gastrointestinal symptoms such as diarrhea, nausea, and abdominal pain. More serious side effects can include liver damage, increased levels of liver enzymes, and fat accumulation in the liver. Therefore, patients taking lomitapide mesylate are often required to have regular liver function tests.

Regulatory Status

Lomitapide mesylate was approved by the Food and Drug Administration (FDA) in the United States in 2012 and by the European Medicines Agency (EMA) in Europe in 2013. It is classified as an orphan drug, a designation given to drugs that treat rare diseases.

See Also

• Familial hypercholesterolemia
• Cholesterol
• Orphan drug