The European Commission (EC) has extended the approval of Ultragenyx Pharmaceutical’s Evkeeza (evinacumab) to include children aged six months to less than five years with homozygous familial hypercholesterolemia (HoFH).
The decision, which follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in November 2024, makes Evkeeza the first approved treatment for this patient group in the EU. Evkeeza was first approved in the EU in June 2021 for adults and adolescents aged 12 years and older with HoFH. The indication was expanded in December 2023 to include children aged five to 11 years.
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HoFH is a rare and life-threatening genetic disorder characterised by extremely high levels of low-density lipoprotein cholesterol (LDL-C) from birth. Without effective treatment, patients are at significant risk of early cardiovascular complications and premature death. Standard care for HoFH involves a combination of diet, lipid-lowering therapies, and in some cases LDL apheresis—a procedure that removes cholesterol from the bloodstream. Evkeeza is approved for use in conjunction with diet and other lipid-lowering therapies.
The EC’s decision was supported by model-based extrapolation of efficacy data from adult patients. Analyses predict that children aged six months to less than five years receiving a 15mg/kg dose of Evkeeza every four weeks would achieve similar or greater LDL-C reductions at 24 weeks compared to adults. Additionally, compassionate use data from five children aged one to four years showed clinically meaningful reductions in LDL-C, consistent with results observed in older paediatric patients. The safety profile for this age group is expected to align with that of older children, with no new safety concerns reported.
In the announcement accompanying the approval, Jane Cooper, senior vice president and EMEA region head at Ultragenyx, said: “We are proud to be able to provide children as young as six 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.”
Evkeeza was discovered and developed by Regeneron Pharmaceuticals, which markets the therapy in the US, whereas Ultragenyx is responsible for its development and commercialisation outside the US. Evkeeza generated $3.6m for Ultragenyx in 2023, as per the company’s financials. According to Regeneron’s 2023 financials, the therapy generated $77.3m in the US in the same year. GlobalData forecasts that Evkeeza will make up to $245m in sales by 2030.
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Evkeeza is a monoclonal antibody (mAb) that is delivered by infusion every month. Regeneron also investigated the therapy in pancreatitis, but a Phase II trial (NCT04863014) was terminated in March 2023 due to a “sponsor decision”.
