Lomitapide: a novel drug for homozygous familial hypercholesterolemia

Abstract

Lomitapide (Juxtapid® and Lojuxta®; Aegerion Pharmaceuticals, Inc., MA, USA), an orally administered inhibitor of the microsomal triglyceride transfer protein, inhibits the synthesis and secretion of ApoB‑containing lipoproteins and, thus, reduces plasma levels of low‑density lipoprotein cholesterol (LDL‑C). Lomitapide has been approved for the therapy of homozygous familial hypercholesterolemia patients. After a proof‑of‑concept Phase II trial, lomitapide has been tested in a multinational single‑arm, open‑label, 78‑week, Phase III trial. Lomitapide effectively reduced mean plasma LDL‑C levels by 50% from baseline in 23 adults with homozygous familial hypercholesterolemia over a 26‑week treatment period and this reduction was sustained for an additional 52 weeks of lomitapide treatment. The Phase III trial also demonstrated that 46% of patients (six out of 13) interrupted or reduced the frequency of apheresis treatments because of an important and stable reduction of LDL‑C. Lomitapide was generally well tolerated and the most common adverse events in the Phase III trial were gastrointestinal and hepatic events.

Keywords:

• HeFH
• HoFH
• lipoprotein apheresis
• lomitapide
• trials

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