The Food and Drug Administration (FDA) approved this Thursday a new cholesterol pill from Merck that works differently than statins.
The daily pill, called Lipfendra, is a PCSK9 inhibitor, a class of powerful cholesterol-lowering drugs that until now were only 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 United States have high levels of LDL cholesterol, according to the American Heart Association (AHA). Too much LDL cholesterol can cause plaque to build up in the arteries, increasing the risk of heart disease and stroke.
In a late-stage clinical trial, Merck found that Lipfendra reduced 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 levels from birth—experienced similar reductions.
“It’s a very effective reduction,” said Dr. Steven Nissen, academic director of the Cardiovascular and Thoracic Institute at the Cleveland Clinic.
This is especially true, Nissen noted, when the PCSK9 inhibitor is taken along with a statin.
“Regardless of the degree of reduction achieved with the statin, a greater reduction is obtained by adding a PCSK9 inhibitor to the statin, and vice versa,” he explained.
Almost all of the patients in Merck’s two clinical trials were also taking a statin, and many were also taking another cholesterol-lowering drug, ezetimibe.
Statins work by increasing the activity of liver receptors that remove LDL cholesterol from the blood, Nissen explained, while PCSK9 inhibitors prevent those receptors from being degraded.
In March, a group of leading medical organizations, including the AHA and the American College of Cardiology, released new guidelines calling for more aggressive cholesterol treatment. People without risk factors for heart disease should aim to keep their LDL cholesterol below 100 mg/dL; those at high risk, below 70 mg/dL; and those who suffer from heart disease, below 55 mg/dL.
Nissen said the pill would be suitable for patients who are already taking the highest statin dose they can tolerate, but whose LDL cholesterol levels remain higher than guidelines recommend.
The first PCSK9 inhibitor was approved in 2015. The drug, Amgen’s Repatha, is injectable. Regeneron and Sanofi also make an injectable PCSK9 inhibitor called Praulent.
Injectable medications, which have similar effectiveness to the new pill, are already widely used, Nissen said. However, a pill is a convenient option for people who don’t want to get a shot, he added.
Lipfendra will cost $10.50 a day, or $315 for a month’s supply, according to Merck. The pharmacist hopes that the out-of-pocket cost will be lower.
