FDA approves Amgen's Repatha PCSK9 drug, opening the door to competition with Sanofi's Praluent

It’s official: on Aug. 27, FDA approved Amgen’s Repatha (evolocumab), the highly anticipated PCSK9 anti-cholesterol drug, a few weeks after it had approved Sanofi’s Praluent (alirocumab)—an action for which Sanofi had paid $65 million to another pharma company under an FDA voucher program, in order to speed its approval closer to Amgen’s. Both products have also been approved in Europe.

The drugs, which showed highly promising results in trials earlier this year, have stirred an anxious reaction from the payer community because millions of US patients have high cholesterol, currently treated (mostly) by low-cost generics. And while Praluent and Repatha were approved for a subset of cholesterol patients, payers worry that if physicians choose to prescribe the newly approved products to many high-cholesterol patients, the cost to the US healthcare system would be measured in the tens of billions of dollars.

Amgen took the somewhat unusual step, in its press release touting the approval, of addressing payer concerns: “Amgen is sensitive to the concerns of payers around cost, budget predictability and paying for value," said Anthony Hooper, EVP 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. By partnering with payers to implement these programs, we can help ensure that all appropriate patients who could benefit from Repatha will have access to this important new therapy.”

Even so, Amgen is pricing Repatha at $14,100 annually, very close to Sanofi’s announced price. (That’s the estimated price for biweekly autoinjector administration of Repatha; Amgen says that a higher dosage for monthly injection will be available next year.)

Within the payer community, there is an expectation of step edits or other measures to control access to the new drugs. At the same time, there is a debate within the medical community over how best to characterize “low,” “high” or “appropriate” cholesterol levels; there is some evidence that lower cholesterol (especially LDL or “bad” cholesterol) is healthier across the board.