Lomitapide for the treatment of hypertriglyceridemia

ABSTRACT

Introduction: Severe genetic forms of hypertriglyceridemia carry a risk of life-threatening pancreatitis and lack available effective treatments. Lomitapide is a microsomal triglyceride transfer protein inhibitor currently approved for treatment of homozygous familial hypercholesterolemia that may be useful in the management of severe hypertriglyceridemia.

Areas covered: Published trials of lomitapide that reported plasma triglyceride response were reviewed, as was a case report of a patient with hypertriglyceridemia who was treated for 13 years with lomitapide. ClinicalTrials.gov was also reviewed for any unpublished results and ongoing trials.

Expert opinion: Lomitapide demonstrates effective triglyceride lowering and may be a useful treatment for patients with genetic hypertriglyceridemia and recurrent acute pancreatitis who are refractory to traditional treatment. However, long term hepatic safety may be a concern and direct clinical trial-level data are lacking for this indication.

KEYWORDS:

• Familial chylomicronemia syndrome
• hypertriglyceridemia
• lipoprotein disorders
• microsomal triglyceride transfer protein (MTP)
• pancreatitis

Declaration of interest

RA Hegele has received honoraria for membership on advisory boards and speakers’ bureaus for Aegerion, Amgen, Lilly, Merck, Pfizer, Sanofi and Valeant. 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.

Additional information

Funding

RA Hegele is supported by the Jacob J. Wolfe Distinguished Medical Research Chair, the Martha G. Blackburn Chair in Cardiovascular Research, and operating grants from the Canadian Institutes for Health Research (Foundation award), the Heart and Stroke Foundation of Ontario (G-15-0009214) and Genome Canada through Genome Quebec (award 4530).