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

References

• Pang J, Chan DC, Watts GF. Critical review of non-statin treatments for dyslipoproteinemia. Expert Rev Cardiovasc Ther. 2014;12(3):359–371.
   PubMedGoogle Scholar
• Sjouke B, Hovingh GK, Kastelein JJP, et al. Homozygous autosomal dominant hypercholesterolaemia: prevalence, diagnosis, and current and future treatment perspectives. Curr Opin Lipidol. 2015;26(3):200–209.
   PubMed Web of Science ®Google Scholar
• Iomitapide (Lojuxta). Use only in homozygous familial hypercholesterolaemia, with caution. Prescrire Int. 2015;24(162):176–178.
   PubMedGoogle Scholar
• Giuliani S, Maggi CA, Regoli D, et al. NK-1 receptors mediate the tachykinin stimulation of salivary secretion: selective agonists provide further evidence. Eur J Pharmacol. 1988;150(3):377–379.
   PubMed Web of Science ®Google Scholar
• Benn M, Watts GF, Tybjaerg-Hansen A, et al. Familial hypercholesterolemia in the danish general population: prevalence, coronary artery disease, and cholesterol-lowering medication. J Clin Endocrinol Metab. 2012;97(11):3956–3964.
   PubMed Web of Science ®Google Scholar
• Das G, Rees A. Microsomal triglyceride transfer protein inhibition: a novel treatment for lowering plasma cholesterol. Curr Opin Lipidol. 2014;25(6):471–473.
   PubMed Web of Science ®Google Scholar
• Davidson MH, Ballantyne CM, Jacobson TA, et al. Clinical utility of inflammatory markers and advanced lipoprotein testing: advice from an expert panel of lipid specialists. J Clin Lipidol. 2011;5(5):338–367.
   PubMed Web of Science ®Google Scholar
• Gouni-Berthold I, Berthold HK. Mipomersen and lomitapide: two new drugs for the treatment of homozygous familial hypercholesterolemia. Atheroscler Suppl. 2015;18:28–34.
   PubMed Web of Science ®Google Scholar
• Thompson GR. Managing homozygous familial hypercholesterolaemia from cradle to grave. Atheroscler Suppl. 2015;18:16–20.
   PubMed Web of Science ®Google Scholar
• Dixon DL, Sisson EM, Butler M, et al. Lomitapide and mipomersen: novel lipid-lowering agents for the management of familial hypercholesterolemia. J Cardiovasc Nurs. 2014;29(5):E7–E12.
   PubMed Web of Science ®Google Scholar
• Gouni-Berthold I, Berthold HK. Diagnose und Behandlung der heterozygoten familiären Hypercholesterinämie. 2nd ed. Bremen: Uni-Med;2015.
   Google Scholar
• Hobbs HH, Brown MS, Goldstein JL. Molecular genetics of the LDL receptor gene in familial hypercholesterolemia. Hum Mutat. 1992;1(6):445–466.
   PubMedGoogle Scholar
• Cuchel M, Bruckert E, Ginsberg HN, et al. Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society. Eur Heart J. 2014;35(32):2146–2157.
   PubMed Web of Science ®Google Scholar
• Nordestgaard BG, Chapman MJ, Humphries SE, et al. Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: Consensus Statement of the European Atherosclerosis Society. Eur Heart J. 2013;34(45):3478–90a.
   Web of Science ®Google Scholar
• Roeters van LJ, Averna M, Alonso R. Treating homozygous familial hypercholesterolemia in a real-world setting: experiences with lomitapide. J Clin Lipidol. 2015;9(4):607–617.
   PubMed Web of Science ®Google Scholar
• Perry CM. Lomitapide: a review of its use in adults with homozygous familial hypercholesterolemia. Am J Cardiovasc Drugs. 2013;13(4):285–296.
   PubMed Web of Science ®Google Scholar
• Sirtori CR, Pavanello C, Bertolini S. Microsomal transfer protein (MTP) inhibition-a novel approach to the treatment of homozygous hypercholesterolemia. Ann Med. 2014;46(7):464–474.
   PubMed Web of Science ®Google Scholar
• Wetterau JR, Aggerbeck LP, Bouma ME, et al. Absence of microsomal triglyceride transfer protein in individuals with abetalipoproteinemia. Science. 1992;258(5084):999–1001.
   PubMed Web of Science ®Google Scholar
• Rader DJ, Brewer HB Jr. Abetalipoproteinemia. New insights into lipoprotein assembly and vitamin E metabolism from a rare genetic disease. JAMA. 1993;270(7):865–869.
   PubMed Web of Science ®Google Scholar
• Stein EA, Raal FJ. New therapies for reducing low-density lipoprotein cholesterol. Endocrinol Metab Clin North Am. 2014;43(4):1007–1033.
   PubMed Web of Science ®Google Scholar
• Iqbal J, Hussain MM. Intestinal lipid absorption. Am J Physiol Endocrinol Metab. 2009;296(6):E1183–E1194.
   PubMed Web of Science ®Google Scholar
• Cuchel M, Bloedon LT, Szapary PO, et al. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med. 2007;356(2):148–156.
   PubMed Web of Science ®Google Scholar
• Davis KA, Miyares MA. Lomitapide: a novel agent for the treatment of homozygous familial hypercholesterolemia. Am J Health Syst Pharm. 2014;71(12):1001–1008.
   PubMed Web of Science ®Google Scholar
• Tuteja S, Duffy D, Dunbar RL, et al. Pharmacokinetic interactions of the microsomal triglyceride transfer protein inhibitor, lomitapide, with drugs commonly used in the management of hypercholesterolemia. Pharmacotherapy. 2014;34(3):227–239.
   PubMed Web of Science ®Google Scholar
• Patel G, King A, Dutta S, et al. Evaluation of the effects of the weak CYP3A inhibitors atorvastatin and ethinyl estradiol/norgestimate on lomitapide pharmacokinetics in healthy subjects. J Clin Pharmacol. 2016;56(1):47–55.
   PubMed Web of Science ®Google Scholar
• Reiner Z. Management of patients with familial hypercholesterolaemia. Nat Rev Cardiol. 2015;12(10):565–575.
   PubMed Web of Science ®Google Scholar
• Wetterau JR, Gregg RE, Harrity TW, et al. An MTP inhibitor that normalizes atherogenic lipoprotein levels in WHHL rabbits. Science. 1998;282(5389):751–754.
   PubMed Web of Science ®Google Scholar
• Robl JA, Sulsky R, Sun CQ, et al. A novel series of highly potent benzimidazole-based microsomal triglyceride transfer protein inhibitors. J Med Chem. 2001;44(6):851–856.
   PubMed Web of Science ®Google Scholar
• Funatsu T, Kakuta H, Takasu T, et al. Atorvastatin increases hepatic fatty acid beta-oxidation in sucrose-fed rats: comparison with an MTP inhibitor. Eur J Pharmacol. 2002;455(2–3):161–167.
   PubMed Web of Science ®Google Scholar
• Dhote V, Joharapurkar A, Kshirsagar S, et al. Inhibition of microsomal triglyceride transfer protein improves insulin sensitivity and reduces atherogenic risk in Zucker fatty rats. Clin Exp Pharmacol Physiol. 2011;38(5):338–344.
   PubMed Web of Science ®Google Scholar
• Samaha FF, McKenney J, Bloedon LT, et al. Inhibition of microsomal triglyceride transfer protein alone or with ezetimibe in patients with moderate hypercholesterolemia. Nat Clin Pract Cardiovasc Med. 2008;5(8):497–505.
   PubMedGoogle Scholar
• Cuchel M, Meagher EA, Du Toit Theron H, et al. Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. Lancet. 2013;381(9860):40–46.
   PubMed Web of Science ®Google Scholar
• Taubel J, Sumeray M, Lorch U, et al. Pharmacokinetics and Pharmacodynamics of Lomitapide in Japanese Subjects. J Atheroscler Thromb. 2015. [Epub ahead of print].
   PubMedGoogle Scholar
• Cuchel M, Blom DJ, Averna MR. Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia. Atheroscler Suppl. 2014;15(2):33–45.
   PubMed Web of Science ®Google Scholar
• Averna M, Cefalù AB, Stefanutti C, et al. Individual analysis of patients with HoFH participating in a phase 3 trial with lomitapide: the Italian cohort. Nutr Metab Cardiovasc Dis. 2016;26(1):36–44.
   PubMed Web of Science ®Google Scholar
• Ito MK, Watts GF. Challenges in the diagnosis and treatment of homozygous familial hypercholesterolemia. Drugs. 2015;75(15):1715–1724.
   PubMed Web of Science ®Google Scholar
• Cuchel M, Bloom DJ, Averna MR, et al. Sustained LDL-C lowering and stable hepatic fat levels in patients with homozygous familial hypercholesterolemia treated with the microsomal triglyceride transfer protein inhibitor, lomitapide: results of an ongoing long-term extension study. Annual Meeting of the American Heart Association; 2013 Nov 16–20; Dallas.
   Google Scholar
• Stefanutti C, Blom DJ, Averna MR, et al. The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia - a post-hoc analysis of a Phase 3, single-arm, open-label trial. Atherosclerosis. 2015;240(2):408–414.
   PubMed Web of Science ®Google Scholar
• Goulooze SC, Cohen AF, Rissmann R. Lomitapide. Br J Clin Pharmacol. 2015;80(2):179–181.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. European public assessment report EPAR for Lojuxta (lomitapide), Annex 1, updated 2015. London: European Medicines Agency; 2013.
   Google Scholar
• Sacks FM, Stanesa M, Hegele RA. Severe hypertriglyceridemia with pancreatitis: thirteen years’ treatment with lomitapide. JAMA Intern Med. 2014;174(3):443–447.
   PubMed Web of Science ®Google Scholar
• Milani RV, Lavie CJ. Lipid control in the modern era: an orphan’s tale of rags to riches. J Am Coll Cardiol. 2013;62(23):2185–2187.
   PubMed Web of Science ®Google Scholar
• Rader DJ, Kastelein JJP. Lomitapide and mipomersen: two first-in-class drugs for reducing low-density lipoprotein cholesterol in patients with homozygous familial hypercholesterolemia. Circulation. 2014;129(9):1022–1032.
   PubMed Web of Science ®Google Scholar
• Raal FJ, Honarpour N, Blom DJ, et al. Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial. Lancet. 2015;385(9965):341–350.
   PubMed Web of Science ®Google Scholar
• Gouni-Berthold I, Berthold HK. PCSK9 antibodies for the treatment of hypercholesterolemia. Nutrients. 2014;6(12):5517–5533.
   PubMed Web of Science ®Google Scholar
• Gouni-Berthold I, Berthold HK. Familial hypercholesterolemia: etiology, diagnosis and new treatment options. Curr Pharm Des. 2014;20(40):6220–6229.
   PubMed Web of Science ®Google Scholar