$67.5 million in 2014 for a voucher that let them speed up the FDA review of alirocumab by a few months, a sign that the partners believed those few months—and a slight head start against Amgen—could return to their bottom line more than the outlay. (With the voucher, which is part of an FDA program to encourage drug development for tropical and rare pediatric diseases, Regeneron and Sanofi jumped the alirocumab review ahead of evolocumab by one month.)
Even more bullish than the analysts and companies themselves, but for different reasons, are the drug-purchasing middlemen who have made it their mission to force down prescription drug prices. Test case number one was in hepatitis C, where the middlemen—Express Scripts (NYSE: [[ticker:ESRX]]) and CVS Health (NYSE: [[ticker:CVS]]) are the two largest—played Gilead (NASDAQ: [[ticker:GILD]]) and AbbVie (NYSE: [[ticker:ABBV]]) off each other for lower prices and have boasted of their success.
Now the purchasers, officially known as pharmacy benefit managers, are sounding the alarm over the cost of PCSK9 drugs. In a much-discussed February blog post in Health Affairs, CVS officials said the class of drugs—which in a few years could also include a candidate from Pfizer that’s currently in Phase 3 trials—“will likely be the highest selling class of medications in history.”
EvaluatePharma predicted last September, however, that it would take four full years for alirocumab and evolocumab to reach the $1 billion sales mark.
That wouldn’t be shabby, but it wouldn’t match the hype built up around these two products.
What’s going on? Statins are now generic and cheap, and it seems most of the older people you know are gobbling them like candy. Why do we need more expensive alternatives?
Some background first: Late last year, the U.S. Centers for Disease Control and Prevention reported that in 2011-2012, nearly 28 percent of Americans age 40 and over used a cholesterol-lowering drug. In the same period, 13 percent of Americans 20 and over were found to have high cholesterol.
Yet heart disease is the cause of 1 in 4 American deaths.
It’s not clear PCSK9 inhibitors will make a big dent. Assuming evolocumab and alirocumab are approved, they’ll first be used for the 620,000 Americans with an inherited version of extremely high cholesterol called familial hypercholesterolemia that statins can’t help with. About one million more Americans who don’t have that particular genetic factor still have extreme cholesterol levels that statins can’t reduce enough. Beyond these patient groups, a certain slice of people with high cholesterol—estimates range from 5 to 20 percent—can’t take statins because of side effects that range from muscle pain and damage to liver problems.
These few million people are the obvious future recipients of the new drugs. (Although insurance companies and their purchasers will be vigilant in making sure statin-intolerant people are in fact statin-intolerant: “Pharmacy benefit managers will attempt to limit that number, based on clinical guidelines and expert opinion,” wrote the CVS/Caremark officials in February. “They will likely require laboratory tests to show evidence of muscle inflammation or liver damage before allowing treatment for those with statin intolerance.”)
Beyond those special populations, however, it’s unclear how deeply into a more general population the drugs will penetrate. “The broader question is, ‘Should we be routinely adding PCSK9 inhibitors to statin therapy in everybody to reduce risk?'” said University of Iowa cardiologist Jennifer Robinson, who is leading the study of alirocumab, during an interview at a cardiology conference last month. “I think we’re going to have to wait for the cardiovascular outcome trials.”
And those trials, which both Sanofi/Regeneron and Amgen are running, won’t divulge their data until late 2017 or early 2018.
Let’s dig into the biology a little. Remember all that talk about good cholesterol and bad cholesterol? The bad stuff is LDL, the low-density lipoproteins. When they hang around in the bloodstream, they clog arteries. Our bodies have trouble breaking down LDL, which is a big reason
