Lomitapide for use in patients with homozygous familial hypercholesterolemia: a narrative review

ABSTRACT

Lomitapide is a drug recently approved for the treatment of patients with homozygous familial hypercholesterolemia. In this article we discuss briefly the pharmacology of this drug followed by a comprehensive narrative review of the available preclinical and clinical data on its safety and efficacy. Only data published as full papers are presented, with the exception of one long-term open-label extension study, which is available only in abstract form.

KEYWORDS:

• Lomitapide
• MTP inhibitors
• lipid-lowering drugs
• cholesterol
• familial hypercholesterolemia

Declaration of interest

D Neef has no conflict of interest to declare. HK Berthold has received speaker’s honoraria from Novartis, MSD and Aspen. IG-Berthold has received research grants from Bayer HealthCare and honoraria from Genzyme, MSD, Novartis, NovoNordisk, Pfizer, Ipsen, Bristol-Myers Squibb, Amgen, Sanofi, Aegereon and Otsuka. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.