Microsomal triglyceride transfer protein (MTP) inhibitors
Oral drugs that stop the liver and gut from assembling VLDL and chylomicrons. Reserved for homozygous familial hypercholesterolaemia.
What they are
Lomitapide is the only member. It lowers LDL even when LDL receptors are absent, which is why it is used in homozygous familial hypercholesterolaemia (HoFH).
Mechanism of action
Microsomal triglyceride transfer protein loads triglycerides onto apolipoprotein B inside the endoplasmic reticulum of hepatocytes and enterocytes. Blocking it prevents the assembly and secretion of VLDL (liver) and chylomicrons (gut), so less VLDL is made and less LDL is formed downstream, independent of LDL receptors.
Target: Microsomal triglyceride transfer protein (MTTP)
Drugs in this class
Test yourself
Think of the answer, then tap the question to see it.
Why does lomitapide work in homozygous FH when statins barely do?
It blocks VLDL assembly upstream, so it lowers LDL without needing LDL receptors.
Why does lomitapide cause fatty liver?
Triglycerides that cannot be exported as VLDL accumulate in hepatocytes.
What diet reduces lomitapide's gastrointestinal effects?
A strict low-fat diet.
Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: Lomitapide: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2026).
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Representative model, for educational purposes only.
