What is the role of lomitapide in the treatment of homozygous familial hypercholesterolemia (FH)?

Updated: Oct 04, 2021
  • Author: Mose July, MD, CCD; Chief Editor: Romesh Khardori, MD, PhD, FACP  more...
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Lomitapide (Juxtapid) is a first-in-class microsomal triglyceride transfer protein (MTP) inhibitor. It was approved by the FDA in December 2012 as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce LDLc, TC, apoB, and non-HDLc in patients with homozygous familial hypercholesterolemia. Because lomitapide increases risk of hepatotoxicity, it is only available through a restricted access program. Approval was based on a small trial of 29 patients exposed to lomitapide, with 23 patients exposed to the drug for 1 year, 15 patients exposed for 2 years, and 5 patients exposed for 3 years. At baseline, the mean LDLc in the homozygous FH patients was 336 mg/dL, and this was reduced 50% after 26 weeks of treatment (P < 0.0001). [38]

A literature review by Liu et al reported that in patients with homozygous FH, lomitapide reduces LDLc, total cholesterol, apoB, and triglyceride levels, even without other lipid-lowering treatments, but also decreases HDLc and apoA-1 values. [39]

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