Lomitapide: a novel drug for homozygous familial hypercholesterolemia

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 LDL 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.