With New Data, Amgen Promises Refund If Cholesterol Drug Doesn't Work

Evolocumab and clinical outcomes in patients with CVD

But Amgen is willing to cut a deal with payers who will drop barriers to coverage which have limited Repatha's commercial success to date.

The Food and Drug Administration approved the first PCSK9 inhibitors in 2015, but only for people with heterozygous familial hypercholesterolemia, an inherited disorder that can severely elevate LDL levels, or for patients who have had heart attacks and other serious cardiovascular problems but still cannot reach target LDL levels using statins.

In an interview Thursday, Harper pointed to additional evidence from the study - adjusted for what he calls a "treatment lag" - showing Repatha reduced the risk of heart attack and stroke by 33%. About 80% had already had a heart attack, and the rest had had a stroke or had pain in their legs and feet from narrowed arteries.

The drug inclisiran could help to reduce the risk of heart attacks and stroke related to high cholesterol, researchers said.

But how much society is willing to pay for better treatments has become a key national issue. The rival drug, alirocumab (Praluent), fared about as badly, with $116 million in 2016 sales.

Insurance companies would lose money on the offer, Bach predicted, because they would have to pay to treat dozens of patients to prevent one heart attack or stroke.

The first part of the story has already been proven - lots of trials in various groups have demonstrated powerful reductions in LDL cholesterol following treatment with anti-PCSK9 antibodies, with reductions of more than half routinely observed even among patients with stubbornly high cholesterol resistant to other lipid lowering medications.

The study, also published in the prestigious New England Journal of Medicine, involved testing Repatha on patients in 49 countries, 1,500 of them in Britain. Amgen is ready to take on some risk for Repatha's real-world performance through financial risk-sharing agreements and outcomes-based contracts (refund the cost of Repatha for eligible patients who have a heart attack or stroke). Investors were particularly disappointed that the study did not show the drug reduced deaths, analysts said. Measuring the difference in all of these events was the study's main goal. ( AMGN ) announced results from the Repatha or evolocumab cognitive function trial (EBBINGHAUS) evaluating the impact on cognitive function in 1,974 patients enrolled in the Repatha cardiovascular outcomes study, FOURIER. Kaiser Health News noted that insurers aren't quite sure whether they would cover the drug, citing issues with its price and its then-unproven benefits.

And the FDA approved evolocumab, made by Amgen in Thousand Oaks, California, only for certain patients, such as those with a hereditary condition that causes extremely high levels of LDL. "The [diabetes] signal is there", he said.

Amgen may know that insurers are unlikely to accept their money-back guarantee, Patterson said.

"I can't think of any intervention in cardiovascular medicine that had a treatment effect of 20 percent or more on major cardiovascular events with this kind of safety profile that has not been incorporated into guidelines or changed practice", he said.

The larger reductions in heart attacks and strokes drove the drug's performance on the MACE measure, and on the primary endpoint, which comprised the MACE numbers as well as hospitalization for unstable angina and coronary revascularization procedures, Levy said. About 70 million Americans have high cholesterol and 25 million take statins, Nissen said.

Not necessarily, say analysts, who reckon TMC and Alnylam can learn from experiences with the established drugs for their phase 3 program, which is due to start mid-year, and set themselves up for success.

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