Lojuxta: new treatment for homozygous familial hypercholesterolaemia

Lojuxta (lomitapide) is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, with or without low-density lipoprotein apheresis, in adults with homozygous familial hypercholesterolaemia (HoFH).

Lojuxta (lomitapide) should be taken on an empty stomach as the fat content of a recent meal may adversely affect gastrointestinal tolerability


Lomitapide selectively inhibits intracellular microsomal triglyceride transfer protein (MTP), thereby reducing lipoprotein secretion and circulating concentrations of cholesterol and triglycerides.1


LDL-C lowering

In a phase III open-label study involving 29 adults with HoFH, addition of lomitapide (median dose 40mg daily) to existing lipid-lowering therapy was associated with a 50% reduction in LDL-cholesterol in the 23 patients who completed 26 weeks of treatment (from a mean of 8.7mmol/L [SD 2.9] at baseline to 4.3mmol/L [SD 2.5], p<0.0001). After completion of the 26-week efficacy phase, patients continued to receive lomitapide and entered a 52-week safety phase (weeks 26 to 78) during which investigators were able to modify concomitant lipid-lowering therapies, including LDL apheresis, at their discretion. Significant reductions in LDL-cholesterol were maintained at weeks 56 and 78 (44% and 38%, respectively; both p<0.0001).2

Reductions in other lipid parameters

Significant reductions from baseline to week 26 were also observed for total cholesterol (46%: 11.1mmol/L [SD 3.5] vs 6.1mmol/L [SD 2.9], p<0.0001), apolipoprotein B (49%: 2.6g/L [SD 0.8] vs 1.3g/L [SD 0.7], p<0.0001) and triglycerides (45%: 1.0mmol/L [0.4—2.9] vs 0.5mmol/L [0.1—1.7], p<0.0001) in the 23 patients who completed the efficacy phase.2

Gastrointestinal effects

The most commonly reported adverse events were gastrointestinal, including diarrhoea, nausea, dyspepsia and vomiting.1,2

Elevation of aminotransferase levels to more than five times the upper limit of normal occurred in four patients and was the most serious adverse reaction reported. Levels normalised following dose reduction or temporary interruption of lomitapide.1,2


  1. Lojuxta Summary of Product Characteristics, July 2013.
  2. Cuchel M et al. Lancet 2013; 381: 40–46.

View Lojuxta drug record

Further information: Aegerion

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