The new results-from a trial with more than 27,500 participants-vindicate the concept that inhibiting PCSK9 can control cholesterol and heart-disease risk. The short duration was likely driven by commercial considerations, as evolucumab's owner, Amgen, sought to gain a head-start over Regeneron and Sanofi, who have an essentially identical product candidate, called alirocumab (Praluent™).
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While doctors said they were relieved that Repatha is safe, doctors such as David Rind said they had hoped the study would show that the injectable medication reduces heart attacks and other serious complications by 30 percent or more, given its success in early studies. Since PCSK9 is a protease that breaks down the LDL receptor, itself the major clearance mechanism for cholesterol from the blood, blocking PCSK9 increases the level of LDL receptor and therefore dramatically reduces circulating cholesterol, and in particular the triglyceride-rich lipoproteins, such as LDL, that are implicated in causing cardiovascular disease.
The results should loosen the purse strings of insurers reluctant to pay for a drug with a list price of over $14,000 a year before discounts, given the high cost of treating strokes and heart attacks.
"As demand and use goes up, the insurance companies usually follow", Steinbaum said. An estimated 75% of prescriptions written for Repatha are rejected. That was good enough for the U.S. Food and Drug Administration (FDA), which approved both drugs in 2015.
By 2021, Repatha sales are expected to reach $3.9 billion, according to the consensus estimate compiled by FactSet. Repatha is also known as evolocumab.
"The prices that are actually being paid by payers in the US marketplace today, after the rather large rebating that goes on, are right in the middle of the value range for a product like this".
So far, payers have thrown up utilization barriers to block coverage for a "significant number" of patients who have been prescribed Repatha, Amgen has indicated in filings with the Securities and Exchange Commission.
"These Repatha results are disappointing", said Dr. Ethan Weiss, a cardiologist and associate professor of medicine at the Cardiovascular Research Institute, University of California San Francisco.
"That reduction in risk is meaningful - but no new records on risk reduction were set."Insurance companies have been trying to escape costly Repatha and have asked the company to provide better proof of reduction in heart disease risk. This large difference has resulted in varying assessments and is critical when thinking about the ultimate value proposition of Repatha.
Trials showed Repatha taken alongside statins reduced the cholesterol levels by 60 per cent more than taking statins alone.
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The group which had received evolocumab experienced fewer primary endpoint events, compared to the placebo arm of the study, with 1,344 (9.8 per cent) compared to 1,563 (11.3 per cent) respectively.
Amgen presented the detailed results from EBBINGHAUS at a Late-Breaking Clinical Trials Session at the American College of Cardiology 66th Annual Scientific Session in Washington, D.C. The new study, published in the New England Journal of Medicine, offers some evidence, however, not as big a benefit as some doctors had hoped.
Sales of the PCSK9 inhibitor have been slow to grow, mainly because health insurers and pharmacy benefit managers in the USA have been reluctant to underwrite prescriptions on the strength of its ability to cut LDL cholesterol alone.
But it comes at a hefty price tag of more than $14,000 per year, raising concerns about how many patients could benefit.
In a trial of more than 27,000 patients, researchers found that taking monthly or twice-monthly injections of the medication, called evolocumab, on top of statins could cut cholesterol levels by nearly 60 per cent on average in patients with an underlying risk of cardiovascular disease.
The investigators who conducted the FOURIER study noted Repatha's benefit grows over time.
Although about 5% of patients in the clinical trial had a heart attack or stroke, rates of those problems could be two to three times higher in the real world, where patients are often older and sicker than those in clinical trials, Ofman said. "You don't get to cherry pick the results you like", he said.