by Gary Finnegan: Patients whose high cholesterol cannot be controlled by currently available therapies are set to have a new treatment option.
The European Medicines Agency (EMA) has recommended authorising Repatha (evolocumab) for people who cannot take statins or for whom statins have proven ineffective. The decisions must formally be approved by the European Commission for it to take effect.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) advised that existing therapies should be used where possible and that medication should be used in addition to a healthy diet.
Repatha is also indicated to treat people with homozygous familial hypercholesterolaemia, a rare inherited disorder in which levels of LDL-cholesterol (‘bad cholesterol’) are higher than normal from birth. It is intended for injection under the skin either once every two weeks, or once a month.
High levels of cholesterol in the blood are common risk factors for heart disease, which is the leading cause of death globally.
Repatha is the first monoclonal antibody in this therapeutic area. It blocks the PCSK9 protein, which would otherwise lower the number of LDL-receptors in the liver and through this, diminishes its ability to remove LDL-cholesterol from the blood.
The efficacy of this new therapy was assessed in nine trials (about 5,500 people) in patients with hypercholesterolaemia and mixed dyslipidaemia, and in two studies (about 250 people) in patients with homozygous familial hypercholesterolaemia. Repatha reduced LDL-cholesterol for both patient groups.
The CHMP also looked at safety information from patients with hypercholesterolaemia and mixed dyslipidaemia, looking at data from over 6,000 patients followed for at least six months and over 1,100 patients followed for at least two years. The Committee considered that the safety profile of Repatha is acceptable, with few patients discontinuing treatment or showing serious adverse events.
A similar safety profile was observed in patients with homozygous familial hypercholesterolaemia. Further data will be collected to assess the implications of very low cholesterol levels.