Inclisiran for treating heterozygous and homozygous familial hypercholesterolaemia in adolescents aged 12 to 17 years

Inclisiran is currently in development for the treatment of heterozygous and homozygous familial hypercholesterolaemia in adolescents aged 12 to 17 years. Familial hypercholesterolaemia is an inherited condition where differences in certain genes affect the liver’s ability to process cholesterol. This can lead to premature cardiovascular diseases. Heterozygous familial hypercholesterolaemia occurs when one gene for familial hypercholesterolaemia is inherited from a single parent, while homozygous familial hypercholesterolaemia is inherited from both parents. Familial hypercholesterolaemia is associated with complications such as ischaemic heart disease, peripheral vascular disease, acute coronary syndrome, stroke and erectile dysfunction. If not treated, people with familial hypercholesterolaemia are at a greater risk of dying from heart disease. Currently, children with familial hypercholesterolaemia have limited treatment options available to them.