Evkeeza by Ultragenyx approved in EU for young children with rare cholesterol disorder

The European Commission (EC) has expanded the authorization for Ultragenyx Pharmaceutical's drug, Evkeeza (evinacumab), to encompass young children from six months to under five years who have homozygous familial hypercholesterolemia (HoFH). This decision makes Evkeeza the first treatment approved in the European Union (EU) for this young demographic suffering from the condition. Initially, Evkeeza was approved in the EU in June 2021 for treating adults and youths aged 12 and over with HoFH, and later in December 2023, the approval was extended to include children aged five to 11.

HoFH is a rare genetic disorder that poses a serious health risk, characterized by extraordinarily high levels of low-density lipoprotein cholesterol (LDL-C) from birth. Those afflicted with this condition face a significant threat of early-onset cardiovascular diseases and premature mortality without appropriate treatment. Generally, managing HoFH involves a combination of dietary modifications, the use of lipid-lowering medications, and sometimes LDL apheresis, a method for removing cholesterol from the bloodstream. Evkeeza is approved for use alongside diet and other lipid-lowering treatments.

The EC’s decision relied on model-based extrapolation from efficacy data seen in adult patients. Predictions suggest that children between six months and less than five years old receiving a 15mg/kg dose of Evkeeza every four weeks could achieve similar or even superior LDL-C reductions over 24 weeks compared to adults. Furthermore, data from compassionate use in five children aged one to four demonstrated clinically significant reductions in LDL-C levels, mirroring results observed in older pediatric groups. The safety profile for these young children is expected to be consistent with that of older children, with no new safety issues identified.

Jane Cooper, the senior vice president and head of the EMEA region at Ultragenyx, expressed pride in the company’s ability to offer a treatment option for very young children living with HoFH, aiming for a significant shift in disease management with Evkeeza’s approval. This advancement represents a major step forward in addressing the needs of this vulnerable patient group.

Evkeeza was originally discovered and developed by Regeneron Pharmaceuticals, which commercializes the therapy within the United States. Outside the US, Ultragenyx handles the development and marketing of Evkeeza. Financial reports indicate that Evkeeza generated $3.6 million in revenue for Ultragenyx in 2023, while in the US, Regeneron recorded $77.3 million in sales for the same year. Projections from GlobalData anticipate that Evkeeza's sales will reach approximately $245 million by 2030.

Evkeeza functions as a monoclonal antibody (mAb) delivered through a monthly infusion. Regeneron also explored its potential use in treating pancreatitis; however, a Phase II clinical trial was terminated in March 2023 due to a "sponsor decision."