The Food and Drug Administration (FDA) has approved evinacumab-dgnb, a novel treatment, for children as young as one year old with homozygous familial hypercholesterolemia (HoFH), a rare and life-threatening genetic condition causing dangerously high levels of LDL cholesterol. The approval, announced March 22, 2023, marks a significant advancement in treating this challenging disorder in very young patients.
HoFH affects approximately 1 in 300,000 newborns, leading to severely elevated cholesterol levels from birth. Without treatment, individuals with HoFH often experience premature cardiovascular disease, including heart attacks and strokes, frequently before the age of 20. Evinacumab-dgnb offers a new therapeutic option for thes children, working by inhibiting the protein angiopoietin-like 3 (ANGPTL3), which normally reduces the clearance of LDL cholesterol from the blood.
Evinacumab-dgnb’s approval is based on data demonstrating its ability to substantially lower LDL cholesterol levels in pediatric patients with HoFH. A position paper from the European Atherosclerosis Society, published in Eur Heart J in 2014, highlighted the need for improved detection and clinical management of familial hypercholesterolemia, including HoFH. Research, including a 2017 study in N Engl J med, has further elucidated the role of ANGPTL3 in cardiovascular disease and paved the way for therapies targeting this pathway.
The treatment is intended for use alongside other cholesterol-lowering therapies,such as LDL-receptor agonists,and is administered via intravenous infusion. Further data regarding the approval can be found on Pharmacy Times: https://www.pharmacytimes.com/view/fda-approves-evinacumab-dgnb-young-children-with-homozygous-familial-hypercholesterolemia.
