The Food and Drug Administration has approved Praluent, a new medicine to lower cholesterol in people who have established heart disease, for people whose risk of a heart attack or stroke is not being adequately controlled by existing drugs called statins.
It might be a big step in the battle against heart disease – and the approval is a triumph for
“Obviously, the class is one of the biggest developments in cardiology in the last decade, that will allow us to treat patients who have been extremely difficult to treat with high levels of LDL cholesterol,” says Steven Nissen, chairman of cardiology at the Cleveland Clinic. “It’s very promising.”
But it will also be very expensive, say Troy Brennan, the chief medical officer of
Praluent, an injection, lowers low-density lipoprotein, the so-called “bad cholesterol”, by as much as 60%, far more than the statin drugs alone. It is the first drug to work by targeting a protein called PCSK9. Mutations in the gene that makes it can result in patients having lower cholesterol and a dramatically lower lifetime risk of heart attacks. A rival medicine from
Regeneron and its partner
“We don’t want the noise about these drugs to be price,” says Leonard Schleifer, the chief executive and founder of Regeneron. “We have to make sure that people get access to the drug for a fair price if they’re insured, for free if they’re not insured, or at a discount if they’re under-insured.”
Praluent's price is 46% higher than that forecast by Evercore ISI, an investment bank, and it is being approved for a larger group of patients than many Wall Street analysts expected following a meeting convened by the FDA to analyze the data on the drug. Some doctors argued its use should be restricted mainly to patients with FH. But Mark Schoenebaum, Evercore ISI's pharmaceutical analyst, says that investors were initially disappointed because they had hoped the FDA would greenlight the drug for an even broader group. Regeneron shares were down 2.5% in afternoon trading before they were halted pending news.
But doctors may prescribe the drug more widely than the label suggests. A survey of physicians published this morning by Geoffrey Porges, an biotechnology analyst at investment bank Sanford C. Bernstein, found that they would use the drug in 30% to 40% of patients who had already had heart attacks. He wrote that 2020 sales of Praluent could be as high as $4.8 billion.
In the survey, doctors expressed a preference for Praluent. One advantage of Regeneron’s drug is that doctors will be able to start it at a lower, 75 milligram dose.
Schleifer, who is a medical doctor and, incidentally, a billionaire thanks to his Regeneron stake, says that the company expects that as many as 8 million people who don’t have their cholesterol well-controlled could be candidates for Praluent. He emphasized, though, that until larger studies prove the drug prevents heart attacks and strokes patients should always choose statins first. It will have sales representatives a
Insurance companies and pharmacy benefit managers, who help insurers and employers manage drug costs, are going to do their best to restrict use of Praluent, though. Brennan, the CVS Caremark executive, says that he expects patients will need to have medical records documenting that they have already taken statins. Those not taking statins would need a blood test confirming that a statin caused elevations in muscle or liver enzymes, not just complaints that they were achy. "We're not trying to replace statins," says Schliefer, the Regeneron CEO.
Most people who have muscle problems from statins do not have those elevated muscle enzymes, says Nissen, the Cleveland Clinic cardiologist, who is conducting clinical trials on related drugs for both Amgen and Pfizer. He says he has patients who are “desperate” to take a statin but who cannot tolerate it because of muscle pain but do not have elevated muscle enzymes.
