Background: Lomitapide (Juxtapid® in US and Lojuxta® in Europe) is the first developed
inhibitor of the microsomal triglyceride transfer protein (MTP) approved as a novel
drug for the management of homozygous familial hypercholesterolemia (HoFH). It acts by
binding directly and selectively to MTP thus decreasing the assembly and secretion of the
apo-B containing lipoproteins both in the liver and in the intestine.
Aims: The present review aims at summarizing the recent knowledge on lomitapide in the
management of HoFH.
Results: The efficacy and safety of lomitapide have been evaluated in several trials and it has
been shown a reduction of the plasma levels of low-density lipoprotein cholesterol (LDL-C)
by a average of more than 50%. Although the most common side effects are gastrointestinal
and liver events, lomitapide presents generally with a good tolerability and satisfactory patients
compliance. Recently, in Europe, to evaluate the long-term safety and efficacy of lomitapide,
the LOWER registry (ClinicalTrials.gov Identifier: NCT02135705) has been established
in order to acquire informations on HoFH lomitapide-treated patients from “real life”
Furthermore, the observation that lomitapide decreases triglyceride levels may be considered
for patients affected by severe forms of hypertriglyceridemia who undergo recurrent episodes
of pancreatitis and are poor responders to conventional treatment.
Conclusion: Lomitapide represents an innovative and efficacious drug for the treatment of
HoFH. Long-term safety data, treatment of pediatric and pregnant HoFH patients and management
of severe hypertriglyceridemia still require further investigations.