Lomitapide for treating homozygous familial hypercholesterolaemia in children aged 5 to 17 years

Lomitapide is currently in clinical development for the treatment of paediatric patients with homozygous familial hypercholesteraemia (HoFH) receiving stable lipid-lowering therapy. HoFH is a more severe form of familial hypercholesterolaemia, a condition passed down through families, which raises blood low density lipoprotein (LDL) cholesterol to very high levels. Low density lipoprotein cholesterol (LDL-C) is often referred to as ‘bad cholesterol’. Attempts to lower LDL-C levels often require multiple lipid-lowering drugs and LDL apheresis. Despite these therapies, most patients with HoFH do not reach guideline-recommended LDL-C levels.