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 ClinicalTrials.gov was also reviewed for any unpublished results and ongoing trials.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.

• Authors: Amanda J Brahm, Robert A Hegele
• Journal: Expert opinion on investigational drugs (Expert Opin Investig Drugs) Vol. 25 Issue 12 Pg. 1457-1463 (Dec 2016) ISSN: 1744-7658 [Electronic] England
• PMID: 27785928 (Publication Type: Case Reports, Journal Article, Review)

Chemical References

• Anticholesteremic Agents
• BMS201038
• Benzimidazoles

Topics

• Animals
• Anticholesteremic Agents (adverse effects, pharmacology, therapeutic use)
• Benzimidazoles (adverse effects, pharmacology, therapeutic use)
• Female
• Humans
• Hypertriglyceridemia (drug therapy, physiopathology)
• Pancreatitis (drug therapy)
• Treatment Outcome