Two studies showed that adding Evkeeza to other cholesterol-lowering treatments effectively reduced LDL cholesterol levels in the blood of patients with homozygous familial hypercholesterolaemia. However, the long-term benefits for the heart and circulatory system still need to be studied.

Side effects with Evkeeza were acceptable, and most patients could receive prolonged treatment (at least one year) without needing to stop.

The European Medicines Agency, therefore, decided that Evkeeza’s benefits are greater than its risks, and it can be authorised for use in the EU.

Evkeeza has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Evkeeza due to the rarity of the disease. Every year, the Agency will review any new information that becomes available, and this overview will be updated as necessary.