Evinacumab for treating homozygous familial hypercholesterolemia in children

Evinacumab is currently in clinical development for the treatment of homozygous familial hypercholesterolaemia (HoFH) in children aged 5-11 years. HoFH is a rare genetic disease that occurs when two of the familial hypercholesterolaemia (FH) causing genes are inherited, one from each parent, resulting in dangerously high levels of ‘bad’ cholesterol (low-density lipoprotein-cholesterol [LDL-C]). This increases the risk of developing heart disease and life-threatening cardiac events as early as teen years. Without treatment, serious cardiovascular complications can occur. Attempts to lower cholesterol levels often require multiple lipid-lowering drugs and machines that remove cholesterol from the blood. Despite these therapies, most patients with this disorder do not reach guideline-recommended LDL cholesterol levels.