Lomitapide

How it works

It inhibits microsomal triglyceride transfer protein in the liver and intestine, reducing the assembly and secretion of apolipoprotein B-containing lipoproteins and thereby lowering LDL-cholesterol.

Prescribing in practice

• Hepatotoxicity, including transaminase elevation and hepatic fat accumulation, is the key risk, so liver function must be assessed before starting and monitored regularly, and it is contraindicated in significant hepatic impairment and in pregnancy.
• It interacts with CYP3A4 inhibitors and is used within a controlled-access programme; concomitant strong or moderate CYP3A4 inhibitors are contraindicated.
• Gastrointestinal effects are common, and a low-fat diet plus fat-soluble vitamin and essential fatty acid supplementation is advised; consult the SPC before prescribing.

Monitoring

Monitor liver transaminases before initiation, before each dose increase and periodically thereafter, watching for hepatic steatosis and rising enzymes.

Counselling the patient

• Advise taking the dose with water and away from food, and to follow a low-fat diet to reduce gastrointestinal upset.
• Stress the importance of avoiding pregnancy and using effective contraception, and of not drinking more than minimal alcohol.
• Explain that regular blood tests of the liver are essential while on treatment.

Evidence & guidelines

Lomitapide is licensed for homozygous familial hypercholesterolaemia on the basis of LDL-cholesterol lowering; refer to the SPC and current prescribing references.