Lomitapide: a guide to its use in adults with homozygous familial hypercholesterolaemia in the EU

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Lomitapide (Lojuxta® [EU]; JuxtapidTM [USA]), an inhibitor of the microsomal triglyceride transfer protein, reduces lipoprotein secretion, resulting in decreases in plasma lipid levels. In a clinical trial in adults with homozygous familial hypercholesterolemia (a rare, potentially life-threatening, genetic disease), adjunctive treatment with oral lomitapide was associated with 40 % reductions from baseline in mean plasma low-density lipoprotein cholesterol levels over a 26-week treatment period, with reductions being sustained for up to 78 weeks with continued treatment. Lomitapide is associated with a clinically manageable adverse event profile, with gastrointestinal effects being the most commonly reported adverse events.

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