Biopharmaceutical company Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) announced on Monday that the European Commission has extended the approval of Evkeeza (evinacumab) for children aged 6 months and older with homozygous familial hypercholesterolemia (HoFH).

HoFH is a rare, inherited form of high cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age.

Evkeeza is the first medicine approved in the EU for children as young as 6 months old to treat HoFH. The decision followed a positive recommendation from the Committee for Medicinal Products for Human Use.

The efficacy of Evkeeza in this age group was predicted from a model-based extrapolation analysis. Additionally, supportive data for five patients aged between 1 and 4 years 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.

Evkeeza was initially approved for adults and adolescents aged 12 years and older with HoFH in June 2021. The indication was later extended to include children aged 5 to 11 years old.

Evkeeza is currently reimbursed and commercially available in the UK, US, Canada, Italy, Japan, the Netherlands, Spain and Luxembourg. It is also available via early access schemes in 13 other countries including Austria and France.

US biotechnology company Regeneron Pharmaceuticals Inc (Nasdaq:REGN) discovered and developed Evkeeza and commercialises it in the United States. Ultragenyx is responsible for development and commercialisation efforts in countries outside of the US.