Evinacumab for children and adolescents with homozygous familial hypercholesterolemia: a plain language summary

Plain Language SummaryWhat is this summary about?This summary reviews three evinacumab studies (two clinical trials and one computer-based model study with real-world data) in children with homozygous familial hypercholesterolemia (HoFH). Evinacumab 15 mg/kg administered into the vein (an intravenous infusion) every 4 weeks was tested in children (aged 5 to 11 years) in the pediatric study and adolescents (aged 12 to 17 years) in a long-term study. Computer-based models were used to predict how the same dose would work in younger children (aged 6 months to 4 years) by using data from clinical trials. This was later verified with real-world data from six children aged 1 to 4 years.What were the results of these studies?Across the clinical studies, evinacumab decreased ‘bad’ cholesterol (low-density lipoprotein cholesterol, or LDL-C) by around 40 to 50% and was generally well tolerated, with no new safety signals identified. In the computer-based model of children from 6 months to 4 years old, similar LDL-C reductions to those from observed real-world data in children 1 to 4 years old were predicted.What do the results mean?These findings supported the recent approval of evinacumab in children as young as 6 months of age in many countries.