Europe first with approval of Amgen’s cholesterol drug Repatha

Amgen's cholesterol drug Repatha has been given the green light in Europe, making it the first in the new PCSK9 inhibitor class approved anywhere in the world.

Repatha (evolocumab) 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. The approval confirms Repatha's lead over its rival, Sanofi and Regeneron's Praluent (alirocumab), and puts it on track to be a leader in a new multi-billion dollar therapy area.

Analysts believe Repatha can achieve peak annual sales in excess of $2 billion, but overall sales will depend on the price the drug can fetch. Following the resistance to high prices seen in cancer and in hepatitis C in the US, many market experts are anticipating a similar battle over the price of the PCSK9 inhibitors.

Also significant is the fact that Amgen has not yet generated data proving Repatha's benefits in cardiovascular morbidity and mortality.

Unmet need

Repatha has been approved by the European Commission for patients with hard-to-treat high cholesterol. This includes patients with hypercholesterolemia (heterozygous familial and non-familial [HeFH]), patients whose condition is not responding adequately to statins or other lipid-lowering drugs, are statin-intolerant or for whom a statin is contraindicated.

Amgen must convince European doctors that there is an unmet need in these groups beyond statin therapy, which has a very large evidence base and is now largely off-patent and low cost.

The company says more than 60 per cent of high-risk patients in Europe are still unable to adequately lower their LDL-C levels with statins or other lipid-lowering agents. It also cites US government research which says among very high-risk patients, the figure is above 80 per cent.

"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, MD, 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."

The drug is given in the form of a subcutaneous injection, either 140 mg every two weeks or 420mg once a month. Before starting treatment, secondary causes of excess cholesterol and abnormal fat levels should be excluded.

Sanofi's Praluent could gain final European approval as early as next week. This would narrow Amgen's lead, creating competition which could allow Europe's health systems to exert downward pressure on prices for both drugs.