lomitapide

Lomitapide is an oral microsomal triglyceride transfer protein (MTP) inhibitor used to lower levels of atherogenic lipoproteins in people with homozygous familial hypercholesterolemia (HoFH). By inhibiting MTP in enterocytes and hepatocytes, lomitapide reduces the assembly and secretion of apolipoprotein B-containing lipoproteins, including chylomicrons and very low-density lipoprotein (VLDL). This leads to lower circulating LDL cholesterol and non-HDL cholesterol, with triglycerides often decreased as well.

In the United States, lomitapide was approved in 2012 as an adjunct to diet to reduce LDL-C, apoB, non-HDL-C, and triglycerides in adults with HoFH. The indication has since been extended to eligible pediatric patients (typically ages 6 years and older) with HoFH. It is marketed under the brand Juxtapid in some markets.

Lomitapide is taken by mouth once daily in the evening with a meal. Dosing is titrated in 5 mg/day increments to a maximum of 60 mg/day, depending on tolerance and response, and should be tailored under medical supervision.

Safety and monitoring: lomitapide carries a risk of serious liver injury and is available only through a Risk Evaluation and Mitigation Strategy (REMS) program. Baseline and periodic liver function tests are required, with more frequent monitoring during the early months of therapy. Common adverse effects include gastrointestinal symptoms such as diarrhea, nausea, and abdominal pain. The drug interacts with strong CYP3A4 inhibitors and induces; concomitant use with strong CYP3A4 inhibitors should be avoided or require dose reductions and close monitoring. Grapefruit should be avoided.