The European Commission (EC) has expanded the approval of Ultragenyx Pharmaceutical's Evkeeza (evinacumab) for the treatment of children aged 6 months and older with homozygous familial hypercholesterolemia (HoFH). This makes Evkeeza the first medicine approved in the European Union for this pediatric population.
HoFH is a rare, inherited condition characterized by dangerously high levels of low-density lipoprotein cholesterol (LDL-C), often referred to as "bad cholesterol." It affects approximately 1,600 people in the EU and 1 in 300,000 globally. Patients with HoFH are at high risk for premature atherosclerotic disease and cardiac events, even at a young age.
Clinical Efficacy and Safety
The EC's decision was based on a model-based extrapolation analysis that predicted the efficacy of Evkeeza in pediatric patients aged 6 months to less than 5 years. The analysis suggested that this age group would experience a similar or greater reduction in LDL-C at week 24 compared to adults when given a 15 mg/kg dose of the drug every 4 weeks. Supportive data from five HoFH patients aged 1 to 4 years who received Evkeeza via compassionate use also demonstrated a clinically meaningful LDL-C reduction consistent with older pediatric patients in clinical studies.
According to Ultragenyx, the safety profile for this younger age group is expected to align with that of older pediatric patients, with no new safety concerns identified.
Expert Commentary
"For very young children suffering from homozygous HoFH, leading to dangerously high LDL-C levels and early cardiovascular disease, treatment options are limited," stated Albert Wiegman, M.D., Ph.D. and Professor, Department of Paediatrics at Amsterdam University Medical Center. "Evinacumab has demonstrated significant LDL-C reductions in adults, adolescents, and children with HoFH from 5-years onwards. The current label expansion for children younger than 5-years illustrates the potential of this medicine to help them control their LDL-C levels at even earlier age in the course of their disease."
Current Treatment Landscape
Prior to this approval, treatment options for very young children with HoFH were limited. Evkeeza was initially approved in the EU in 2021 for adult and adolescent patients aged 12 years and older with HoFH, and the label was later expanded in 2023 to include children aged 5-11 years old. The drug is also approved in other regions, including the United States, United Kingdom, and Canada.
Commercial Availability
Evkeeza is currently reimbursed and commercially available in the UK, U.S., Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg. It is also available via early access schemes in 13 additional countries, including Austria and France.
About Evkeeza
Evkeeza (evinacumab) is an angiopoietin-like 3 (ANGPTL3) inhibitor. It works by attaching to the ANGPTL3 protein in the body and blocking its effects, which in turn reduces cholesterol levels in the blood. The drug is administered via an infusion every four weeks.
Regeneron Pharmaceuticals discovered and developed Evkeeza and commercializes it in the U.S. Ultragenyx is responsible for the development and commercialization of Evkeeza in countries outside the U.S.
