European Commission Approves Amgen's New Cholesterol-Lowering Medication Repatha (Evolocumab)

The European Commission (EC) has granted marketing authorization for Amgen's Repatha (evolocumab), the first proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor to be approved in the world, for the treatment of patients with uncontrolled cholesterol who require additional intensive low-density lipoprotein cholesterol (LDL-C) reduction.

Repatha is a human monoclonal antibody that inhibits PCSK9, a protein that reduces the liver's ability to remove LDL-C, or 'bad' cholesterol, from the blood. Elevated LDL-C is an abnormality of cholesterol and/or fats in the blood and is recognized as a major risk factor for cardiovascular disease (CVD).

The EC approved Repatha for

•  The treatment of adults with primary hypercholesterolemia (heterozygous familial and non-familial [HeFH]) or mixed dyslipidemia, as an adjunct to diet:
     •  in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin, or
     •  alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.
•  The treatment of adults and adolescents aged 12 years and over with homozygous familial hypercholesterolemia (HoFH) in combination with other lipid-lowering therapies.

The effect of Repatha on cardiovascular morbidity and mortality has not yet been determined.

More than 60% of high-risk patients in Europe are still unable to adequately lower their LDL-C levels with statins or other currently approved lipid-lowering agents. Among very high-risk patients, the percentage is increased to more than 80%. The health care cost of CVD in the EU is approximately €106 billion per year.

"We are proud that our cholesterol-lowering medication, Repatha, is the first PCSK9 inhibitor to be approved by any regulatory agency in the world," said Sean E. Harper, executive vice president of Research and Development at Amgen. "High LDL cholesterol is a major global health burden and many patients are unable to appropriately control their LDL cholesterol with the maximum tolerated dose of a statin, or are unable to take statins due to intolerance or contraindications. We are excited to make this new cholesterol-lowering medication available for patients in Europe."

One high-risk patient group includes those with familial hypercholesterolemia (FH), an inherited condition caused by genetic mutations which lead to high levels of LDL-C at an early age. It is estimated that less than 1% of people with FH (heterozygous and homozygous forms) in most countries are diagnosed.

"Many patients who are taking cholesterol-lowering therapies, including those with familial hypercholesterolemia, still struggle to control their LDL cholesterol levels," said John J.P. Kastelein, professor of medicine and chairman of the Department of Vascular Medicine at the Academic Medical Center (AMC) of the University of Amsterdam. "As the first in a new class of drugs in the EU, evolocumab will offer physicians an important and innovative treatment option for patients with uncontrolled cholesterol who require additional LDL cholesterol reduction."

Approval from the EC grants a centralized marketing authorization with unified labeling in the 28 countries that are members of the EU. Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions on the basis of the decision of the EC.