Amgen's cholesterol-lowering PCSK9 inhibitor Repatha cleared in the US

The FDA announced Thursday that it approved Amgen's Repatha (evolocumab) for use in addition to maximally-tolerated statin therapy in adults with heterozygous familial hypercholesterolaemia (HeFH), homozygous familial hypercholesterolaemia (HoFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL cholesterol. The approval marks the second PCSK9 inhibitor cleared in the US after Sanofi and Regeneron Pharmaceuticals' Praluent (alirocumab) last month for patients with HeFH and those with ASCVD needing additional lowering of LDL cholesterol.

Repatha's approval, which follows a recent recommendation by an FDA advisory panel, was supported by data from a 52-week placebo-controlled trial and eight 12-week placebo-controlled trials of patients with primary hyperlipidaemia, including two studies that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In study data published earlier this year in the NEJM, both Repatha and Praluent were found to cut the risk of cardiovascular events by about half and lower LDL cholesterol levels by more than 60 percent.

Amgen said Repatha will carry a US wholesale price of just over $542 per single-use injection, or $14 100 a year for bi-weekly administration. The company indicated that a monthly formulation would be made available next year.

Earlier this month, the CVS Health Research Institute argued that current guidelines for managing high cholesterol should be revisited in light of the introduction of "high-cost" PCSK9 inhibitors. CVS Health previously estimated that PCSK9 inhibitors could increase healthcare spending in the US by as much as $150 billion a year (for additional analysis, see ViewPoints: Feeling heat from PCSK9s, CVS reignites debate on cholesterol targets).

Anthony Hooper, executive vice president of global commercial operations at Amgen, said the company "is sensitive to the concerns of payers around cost, budget predictability and paying for value." He indicated that Amgen "will be working with payers and other purchasers to provide innovative pricing programmes linking the net price of Repatha to the expected LDL-cholesterol reductions and anticipated appropriate patient utilisation," adding that "by partnering with payers to implement these programmes, we can help ensure that all appropriate patients who could benefit from Repatha will have access to this important new therapy."

Last month, Repatha became the first PCSK9 inhibitor to be cleared anywhere in the world following its approval in Europe. Meanwhile, Praluent remains under review in Europe after the European Medicines Agency's Committee for Medicinal Products for Human Use backed the therapy in July. Analysts have estimated that PCSK9 inhibitors could amass $10 billion in annual revenue.

For related analysis see ViewPoints: Amgen pays lip service to Repatha pricing concerns – but will it follow through?

For further analysis on the PCSK9 inhibitor class, see Physician Views Poll Results: How do doctors see Praluent and Repatha stacking up after FDA panel meetings? and ViewPoints: Regeneron, Sanofi seek leverage via Praluent's aggressive US pricing.

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