ANGPTL3 inhibitors
Drugs that neutralise ANGPTL3, a liver protein that brakes lipases. They lower LDL without needing LDL receptors, so they help homozygous FH.
What they are
Evinacumab, a monoclonal antibody given by monthly infusion, is the member in use. It is added to other lipid-lowering treatment in homozygous familial hypercholesterolaemia, where statins and PCSK9 inhibitors work poorly because LDL receptors are absent or barely work. Lomitapide (an MTP inhibitor) is the other LDL-receptor-independent option in this disease.
Mechanism of action
ANGPTL3 (angiopoietin-like protein 3), made in the liver, inhibits lipoprotein lipase and endothelial lipase. Neutralising it frees these lipases, so triglyceride-rich lipoproteins are broken down faster and less LDL is formed. LDL cholesterol falls by a route that does not depend on LDL receptors.
Target: Angiopoietin-like protein 3 (ANGPTL3)
Drugs in this class
Test yourself
Think of the answer, then tap the question to see it.
Which enzymes does ANGPTL3 normally inhibit?
Lipoprotein lipase and endothelial lipase.
Why do ANGPTL3 inhibitors work in homozygous FH?
They lower LDL by a pathway that does not need LDL receptors.
What is the main acute risk during an evinacumab infusion?
Infusion reactions and serious allergic reactions, including anaphylaxis.
Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: Evinacumab-dgnb Injection: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2025); ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026).
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Representative model, for educational purposes only.