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lomitapide
- Development stage
- Approved
- Lead developer
- Chiesi
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Lomitapide is a small molecule antihyperlipidemic agent used as an adjunct to a low-fat diet and other lipid-lowering treatments for the management of homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder characterized by extremely high cholesterol levels. Lomitapide acts by directly inhibiting microsomal triglyceride transfer protein (MTP) in the endoplasmic reticulum, which prevents the formation of apolipoprotein B-containing lipoproteins such as very low-density lipoprotein (VLDL) and chylomicrons. This inhibition leads to reduced production of low-density lipoprotein cholesterol (LDL-C) and total cholesterol in the blood[1][2][3][4][7]. Lomitapide is available only through restricted distribution programs due to its risk profile, including potential liver toxicity[1][2].
- Brand names
- JuxtapidLojuxta
- Other names
- lomitapideAEGR-733AEGR733AEGR 733
02
Targets
MTTP (Microsomal triglyceride transfer protein large subunit)
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