The FDA approved Amgen’s Repatha, an injectable PCSK9 inhibitor that controls control low-density lipoprotein (LDL) cholesterol. Repatha has been approved for treatment in patients with the genetic conditions heterozygous familial hypercholesterolemia (HeFH) and homozygous familial hypercholesterolemia (HoFH). Repatha has also been approved for use in patients with clinical atherosclerotic cardiovascular disease. 

A clinical trial found Repatha lowered LDL cholesterol by 60 percent in patients with HeFH who had previously used other lipid lowering therapies. Repatha blocks PCSK9, a liver protein that lowers the number of receptors on the liver that remove LDL cholesterol from the bloodstream, therefore reducing overall LDL cholesterol levels.

"Amgen is sensitive to the concerns of payers around cost, budget predictability and paying for value," said Anthony C. Hooper, executive vice president of Global Commercial Operations at Amgen. "We are confident in the ability of Repatha to demonstrate real-world effectiveness and value based on intensive LDL cholesterol reductions, and we will be working with payers and other purchasers to provide innovative pricing programs linking the net price of Repatha to the expected LDL cholesterol reductions and anticipated appropriate patient utilization."

Repatha is the second approved PCSK9 inhibitor marketed today. It will compete with Praluent, marketed by Sanofi and Regeneron, for market share in the treatment of patients with HeFH. (Sanofi and Regeneron did not seek approval for Praulent in HoFH cases.) Amgen’s PCSK9 inhibitor will also compete with Aegerion's Juxtapid for HoFH treatment. 

Repartha reportedly will cost $14,100 per year before discounts, or about $500 less than Praulent’s annual pre-discount price.

Repartha is expected to be available this week. 

Our Take: High levels of LDL cholesterol is linked to heart disease—the leading cause of death in the US. PCSK9 inhibitors like Repatha are a major breakthrough in treating high levels of LDL cholesterol, especially when statins are insufficient. 

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