European Commission extends Evkeeza approval to children as young as 6 months with HoFH

The European Commission has extended the approval of Evkeeza (evinacumab) to include children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), an ultrarare inherited condition characterized by dangerously high levels of low-density lipoprotein cholesterol (LDL-C).

This decision makes Evkeeza the first medication in the European Union approved for treating HoFH in this young age group.

Previously, Evkeeza was approved for patients aged 5 years and older.

Efficacy predictions, based on model-based extrapolation analyses, suggest that children aged 6 months to less than 5 years will experience similar or greater reductions in LDL-C levels compared to adults when administered a 15 mg/kg dose every 4 weeks.

Supportive data from compassionate use in 5 patients aged 1 to 4 years indicate clinically meaningful LDL-C reductions consistent with those observed in older patients.

No new safety concerns have been identified in this younger population.

Read the full press release here.