European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

Core News Summary - The European Commission (EC) has extended the approval of Evkeeza (evinacumab) for treating children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH), making it the first and only medicine approved in the EU for this age group [1][2] - Evkeeza is an ANGPTL3 inhibitor that reduces LDL-C levels and is now indicated for pediatric patients as young as 6-months old, addressing a critical unmet need in managing HoFH [1][5] Efficacy and Safety - Model-based extrapolation analysis predicts that pediatric patients aged 6-months to less than 5 years will experience a similar or higher magnitude of LDL-C reduction at week 24 compared to adults when receiving a 15 mg/kg dose every 4 weeks [2] - Compassionate use data for five patients aged 1-4 years showed clinically meaningful LDL-C reductions consistent with older patients, with no new safety concerns identified [2] Market and Availability - Evkeeza is now reimbursed and commercially available in the UK, US, Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg, with early access schemes in 13 additional countries including Austria and France [2] - The treatment was initially approved for adults and adolescents aged 12+ in June 2021, with the indication extended to children aged 5-11 in December 2023 [2] Disease Background - HoFH is an ultrarare genetic disorder affecting 1 in 300,000 people globally and approximately 1,600 in the EU, characterized by dangerously high LDL-C levels (>400 mg/dL) and early cardiovascular risks [3][4] Product Mechanism - Evkeeza works by blocking the ANGPTL3 protein, which regulates cholesterol levels, and is administered via monthly infusions [5] Development and Commercialization - Evkeeza was discovered and developed by Regeneron Pharmaceuticals, Inc, with Ultragenyx responsible for its development and commercialization outside the US [7] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for rare and ultrarare genetic diseases, with a diverse portfolio addressing high unmet medical needs [12][13]