Federal regulators on Friday approved the first of a new class of drug that can sharply lower cholesterol levels, offering a new option for millions of Americans suffering from cardiovascular disease, the nation’s leading killer.
But the drug, Praluent, which analysts project will become a huge seller, is expected to become the next flashpoint in the growing controversy of escalating pharmaceutical prices, and health plans are expected to put in place strict measures to control which patients can use the drug and prevent it from becoming a budget buster.
The list price of Praluent is about $14,600 a year, substantially higher than the $7,000 to $12,000 that some health plan executives and Wall Street analysts had been expecting. Typically insurers and government health programs get discounts or rebates.
Sanofi and Regeneron Pharmaceuticals, which developed the product, said the price was justified by the potential benefits to patients and savings to the health care system that the drug would provide by preventing heart attacks and strokes — though the ability of the drug to do that has not been proved.
“We came to a price that is reflective of value, not what the market will bear,” said Elias Zerhouni, head of research and development at Sanofi, who said his own brother had suffered three heart attacks and needed new options to control cholesterol.
In clinical trials, Praluent reduced levels of LDL cholesterol, the so-called bad cholesterol, by 40 percent or more, even among patients already taking statins, the mainstay pills like Lipitor for controlling blood lipids. Some cardiologists say Praluent and similar drugs in the pipeline represent significant advances.
“I can tell you there is a lot of hope on the part of our patients,” said Dr. Seth Martin, associate director of the lipid clinic at Johns Hopkins University. “There is such a high need for these medications.”
Praluent, also known as alirocumab, was endorsed last month by outside advisers to the Food and Drug Administration. So was Repatha, or evolocumab, a similar drug developed by Amgen that is expected to win F.D.A. approval by the end of August.
But there was considerable debate among the advisers as to how broadly the drugs should be used. In its decision on Friday, the F.D.A. approved Praluent for patients who have had heart attacks, strokes, chest pain or related conditions, or have a genetic condition that causes high cholesterol and who require additional lowering of LDL despite taking the highest dose of a statin that they can tolerate.
Executives at Sanofi and Regeneron estimate there are eight million to 10 million Americans in those categories who need to further lower their cholesterol. But they said they did not know how many of them were already taking a maximally tolerated dose of statins.
Regulators in Europe, where Repatha was approved and Praluent recommended for approval this week, will allow broader use, like by people who have high risk but have not yet had a heart attack or stroke.
Studies aimed at showing that the drugs prevent heart attacks and strokes are underway, but results are not expected until about 2017. Some doctors say they will use the drugs sparingly or not at all until then.
“This is treating a lab value,” said Dr. Rita Redberg, a cardiologist at the University of California, San Francisco, referring to lowering cholesterol for its own sake. “I don’t think we should rush into it.”
Some doctors are also worried about possible long-term safety, though the clinical trials did not show significant problems.
Health plans are worried that so many patients might use the drugs, which might be taken for life, that it would cost billions or even tens of billions of dollars a year.
The health care system is still smarting from the experience with Sovaldi, a rapidly adopted drug for hepatitis C from Gilead Sciences that cost $1,000 a pill. Gilead sold $12.4 billion worth of Sovaldi and a related drug in 2014, the first full year on the market, straining the budgets of insurance companies and Medicaid programs.
Lessons learned, payers appear much better prepared for Praluent. “We will limit it to those select individuals at the highest risk,” said David Lassen, chief clinical officer at Prime Therapeutics, a pharmacy benefit manager.
One way health plans will do that is to keep patients on statins, which are mainly inexpensive generics.
So the new drugs could improve public health indirectly, by increasing use of statins, which are proved to prevent heart attacks. “We’re going to help some people without even taking our drug,” said Leonard S. Schleifer, chief executive of Regeneron.
Dr. Troyen Brennan, chief medical officer of CVS Health, which owns a pharmacy benefit manager, said his company would demand evidence through blood tests if a patient claimed to be unable to tolerate statins because of side effects and wanted to switch to one of the expensive new drugs. Dr. Brennan said he was disappointed in the price set by the companies.
Express Scripts, the largest pharmacy benefits manager, has told its clients that it would switch patients getting statins to mail delivery because patients served this way were less likely to quit taking their medicines than those who pick up prescriptions at the drugstore.
Pharmacy benefit managers and health plans might also pit Amgen and Sanofi against each other, choosing to use only the least expensive drug and shut out the other. That tactic worked well to wring discounts from Gilead, once it faced competition in hepatitis C from AbbVie starting late last year.
The new drugs are called PCSK9 inhibitors because they stifle a protein with that name that is involved in cholesterol regulation. The development of the drugs stemmed from the discovery that people with genetic mutations that lowered the activity of the protein have very low cholesterol and low cardiovascular risk. Pfizer and other companies are also developing such drugs.
Praluent would be self-injected every two weeks and comes in two available doses. The list price is $560 per injection for either dose, equivalent to $40 a day or $14,600 a year.
Crestor, a brand name statin, costs less than $3,000 a year. Dr. Schleifer of Regeneron said it was unfair to compare the price of statins with that of Praluent, which will be used for fewer patients. Praluent is also a monoclonal antibody, a protein made in genetically engineered living cells. Those drugs are more expensive to manufacture than pills like statins.
Dr. Zerhouni of Sanofi said there would not be a repeat of what happened with Sovaldi.
“Gilead shocked the system by $12 billion a year the first year,” Dr. Zerhouni said. “In our wildest dream we don’t see that happening” with the cholesterol drugs.
Wall Street analysts estimate sales of several hundred million dollars for Praluent in 2016, though eventually sales could climb to several billion dollars annually.
Among the early users might be adults with so-called heterozygous familial hypercholesterolemia, an inherited condition that causes extremely high cholesterol and a substantially increased risk of heart attacks. Estimates are that at least 600,000 Americans have this condition.
“It represents a new era of hope for us,” said Katherine Wilemon, founder and president of the FH Foundation, an advocacy group. “We tend to take everything out there that is available, and even with that we can’t get our LDL levels down to a ‘safe’ level.”
Ms. Wilemon, whose organization gets contributions from drug companies, suffered a heart attack in 2006, at age 39. About nine months ago, even though she was taking a statin and other drugs, her LDL level was about 167, much higher than the 70 or 100 that would be recommended for her. But when she began taking one of the new drugs in a study, her LDL levels dropped below 50.
Correction: July 24, 2015
Because of an editing error, an earlier version of this article rendered incorrectly the name of the government agency that approved the new drug. It is the federal Food and Drug Administration, not the Federal Drug Administration.
Correction: July 24, 2015
An earlier version of this article incorrectly referred to a type of cholesterol drug. It is a PCSK9 inhibitor, not a PSCK9 inhibitor.