Evkeeza

Evkeeza can only be obtained with a prescription, and treatment should be started and supervised by a doctor experienced in the treatment of lipid disorders (altered fat levels in the blood). Patients should be on stable doses of other cholesterol-lowering treatments before they can start treatment with Evkeeza.

Evkeeza is given as an infusion (drip) into a vein for 60 minutes every four weeks.

For more information about using Evkeeza, see the package leaflet or contact your doctor or pharmacist.

The active substance in Evkeeza, evinacumab, is a monoclonal antibody (a type of protein) that has been designed to attach to ANGPTL3, a protein that blocks certain lipases (enzymes that break down fats) in the body. Once evinacumab attaches to ANGPTL3, the lipases can function again, which lowers the blood levels of fats and reduces cholesterol.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Evkeeza have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Evkeeza are continuously monitored. Side effects reported with Evkeeza are carefully evaluated, and any necessary action is taken to protect patients.

Since Evkeeza has been authorised under exceptional circumstances, the company that markets Evkeeza will provide results every year, highlighting the medicine’s long-term safety, outcomes of any pregnancies that occur, and the effect on fatty deposits in the arteries (atherosclerosis). The company that markets Evkeeza will collect these results from an ongoing registry (a collection of information) of patients with homozygous familial hypercholesterolaemia.

A main study showed that Evkeeza effectively reduced LDL cholesterol levels in adults and adolescents aged 12 years and older with homozygous familial hypercholesterolaemia. The participants received Evkeeza or placebo (a dummy treatment) while also taking other cholesterol-lowering therapies.

The study involved 65 patients who received either Evkeeza or placebo once every four weeks. After 24 weeks, the average LDL cholesterol levels in the blood of patients receiving Evkeeza had reduced by around 47% from the start of the treatment, compared with about a 2% increase in patients receiving placebo. The improvement in LDL cholesterol levels with Evkeeza was maintained when treatment was given for 24 additional weeks.

An additional main study looked at the effectiveness of Evkeeza in 14 children aged 5 to 11 years with homozygous familial hypercholesterolaemia. In this study, Evkeeza was not compared with another treatment or placebo. After 24 weeks of treatment, Evkeeza lowered LDL cholesterol levels by 48%.

A study in 6 children aged 5 to 11 years showed that Evkeeza acts in the same way in the body of younger children as in that of adolescents and adults.

The benefits of Evkeeza in children aged from 6 months to less than 5 years were determined following an analysis of data of how the medicine behaves in the body. This included data from older children and adults, along with assumptions about young children's development and the impact of having homozygous familial hypercholesterolaemia. Results indicated that, although children in this age group have lower blood levels of Evkeeza at the recommended doses, the medicine’s effectiveness in treating homozygous familial hypercholesterolaemia is comparable to that seen in adults and older children. These findings were supported by data from five children who started treatment with Evkeeza before the age of 5 through a compassionate use programme.

Two studies showed that adding Evkeeza to other cholesterol-lowering treatments effectively reduced LDL cholesterol levels in the blood of adults and children from 12 years of age with homozygous familial hypercholesterolaemia. A third study in children aged 5 to 11 years showed comparable results. The Agency also considered that the effectiveness of Evkeeza in children aged from 6 months to less than 5 years is comparable to that of older children and adults. However, the long-term benefits of treatment 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.

Although uncertainties remain, 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. The company must provide further data on Evkeeza. It must submit data regarding the medicine’s long-term safety, outcomes of any pregnancies that occur, and the effect of treatment on fatty deposits in the arteries (atherosclerosis). The company will collect these data from an ongoing registry (a collection of information) of patients with homozygous familial hypercholesterolaemia. Every year, the Agency will review any new information that becomes available, and this overview will be updated as necessary.

For the full list of side effects and restrictions of Evkeeza, see the package leaflet.

The most common side effect with Evkeeza (which may affect more than 1 in 10 people) is inflammation of the nose and throat. Other side effects (which may affect up to 1 in 10 people) include flu-like illness, dizziness, back pain and nausea (feeling sick). The most frequent serious side effect (which may affect up to 1 in 100 people) is anaphylaxis (a sudden, severe allergic reaction).

Evkeeza received a marketing authorisation valid throughout the EU on 17 June 2021.