The Food and Drug Administration on Thursday approved a new cholesterol pill from Merck that works in a different way from statins.
The daily pill, called Lipfendra, is known as a PCSK9 inhibitor — a class of powerful cholesterol-lowering medication that had previously only been available as injections.
The drug was approved for people with hypercholesterolemia, or high levels of LDL cholesterol, the so-called bad cholesterol.
About 1 in 4 adults in the U.S. have high LDL cholesterol, according to the American Heart Association. Too much LDL cholesterol can lead to plaque buildup in the arteries, increasing the risk of heart disease and stroke.
In one late-stage clinical trial, Merck found that Lipfendra lowered LDL cholesterol levels by up to 60% after 24 weeks. In a second trial, people with a genetic condition called heterozygous familial hypercholesterolemia — which causes sky-high cholesterol from birth — saw similar reductions.
“It’s a very effective reduction,” said Dr. Steven Nissen, the chief academic officer of the Heart Vascular and Thoracic Institute at the Cleveland Clinic.
That’s especially the case, Nissen said, when the PCSK9 inhibitor is taken alongside a statin.
“Whatever amount of lowering you get from the statin, you get more lowering by adding a PCSK9 on top of the statin, and vice versa,” he said.
Nearly all of the patients in the two Merck clinical trials were also taking a statin, and many were also taking a different cholesterol-lowering drug, ezetimibe.
Statins work by increasing the activity of receptors in the liver that pull LDL cholesterol out of the blood, Nissen said, while the PCSK9 inhibitors prevent those receptors from degrading.
In March, a group of major medical organizations, including the AHA and the American College of Cardiology, released new guidelines urging more aggressive treatment of cholesterol. People without heart disease risk factors should aim to get their LDL cholesterol below 100 mg/dL, people at high risk should aim for under 70 mg/dL and for people with heart disease, under 55 mg/dL.
Nissen said the pill would be good for patients who are on the highest dose of a statin that they can tolerate, but whose LDL cholesterol levels are still higher than the guidelines recommend.
The first PCSK9 inhibitor was approved in 2015. The drug, Repatha from Amgen, is an injectable. Regeneron and Sanofi also make an injection PCSK9 inhibitor called Praulent.
The injectable drugs, which are similarly effective to the new pill, are already commonly used, Nissen said. But a pill is a convenient option for people who don’t want an injection, he added.
Lipfendra will cost $10.50 a day, or $315 for a 30-day supply, according to Merck. The drugmaker expects the out-of-pocket cost will be lower.
