Ultragenyx receives EC decision for Evkeeza expanded indication

Ultragenyx Pharmaceutical announced that the European Commission has extended the approval of Evkeeza as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia. Evkeeza is the first angiopoietin-like 3 inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol caused by HoFH. Evkeeza initially received EC decision as an adjunct to diet and other lipid-lowering therapies in adolescents and adults aged 12 years and older with HoFH in June 2021. This EC decision follows the positive recommendation from the Committee for Medicinal Products for Human Use received in November 2023 and is based on the results of a Phase 3 open-label study in patients ages 5-11 years with HoFH.1 Despite treatment with other lipid-lowering therapies, children entered the Phase 3 trial with an average LDL-C level of 264 mg/dL, more than twice the target for paediatric patients with HoFH. With the addition of Evkeeza, children were able to reduce their LDL-C by 48% at week 24 on average. Significant reductions were also observed in other key secondary endpoints including levels of apolipoprotein B, non-high-density lipoprotein cholesterol and total cholesterol. The safety profile of Evkeeza was with the safety profile observed in adults and paediatric patients aged 12 years and older, with the additional adverse reaction of fatigue.