Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, announced that the European Commission (EC) has extended the approval of Evkeeza (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an angiopoietin-like 3 (ANGPTL3) inhibitor, is the first medicine indicated for children in the European Union (EU) as young as 6-months old to treat HoFH, a disease associated with dangerously high levels of low-density lipoprotein cholesterol (LDL-C).
“For very young children suffering from homozygous HoFH, leading to dangerously high LDL-C levels and early cardiovascular disease, treatment options are limited. Many young children don’t reach treatment goals, leaving them with an uncertain future,” 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.”
This EC decision follows the positive recommendation received from the Committee for Medicinal Products for Human Use in November 2024.The efficacy of Evkeeza in paediatric patients aged 6 months to less than 5 years with HoFH has been predicted from a model-based extrapolation analysis. Results of these analyses show that paediatric patients aged 6 months to less than 5 years are predicted to experience a similar or higher magnitude of percent change in LDL-C at week 24 compared to adults, when receiving a 15 mg/kg dose every 4 weeks. In addition, supportive data for five patients between 1 and 4 years old with HoFH who received Evkeeza via compassionate use show a clinically meaningful reduction of LDL-C consistent with that observed in patients aged 5 years or older in clinical studies. Based on the currently available data, the safety profile in pediatric patients aged 6-months to 5 years old is expected to be similar to the safety profile in older pediatric patients. No new safety concerns have been identified in the compassionate use program.
“The international community of parents and caregivers of children with HoFH welcomes this approval, because this is a potentially life-changing therapy for the children and their parents affected by this rare and severe disorder,” stated Magdalena Daccord, chief executive officer of FH Europe Foundation. “As we advocate for childhood screening and detection to help improve early HoFH diagnosis, it is key to be able to offer to young patients appropriate and innovative treatment solutions along with lifestyle management. That said, it will be a true success once this therapy option is available to all those who need it as soon as they need it.”
Evkeeza initially received approval as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C)-lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with HoFH in June 2021. The indication was later extended to include children aged 5 to 11 years old with HoFH, with EC decision for that variation received in December 2023. The treatment is now reimbursed and commercially available to prescribe for appropriate patients with HoFH 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.
“HoFH can cause severe cardiovascular events including heart attacks even in young children, making diagnosing it and reducing the high LDL-C levels it causes an urgent matter,” said Jane Cooper, senior vice president, EMEA region head at Ultragenyx. “We are proud to be able to provide children as young as 6-months old living with HoFH the first approved medicine to reduce LDL-C levels and hope that it will support a fundamental shift in the management of the disease.”