FDA clears Merck cholesterol pill targeting PCSK9
Merck’s daily Lipfendra is a PCSK9 inhibitor for high LDL cholesterol, a drug class previously available only as injections.
By Georgia Hale · Staff Writer
2 min read
The Food and Drug Administration on Thursday approved Merck’s Lipfendra, a once-a-day cholesterol pill that attacks LDL cholesterol from a different angle than statins.
The drug is a PCSK9 inhibitor, a class of cholesterol-lowering medicines that, until now, had been given as shots. The FDA cleared Lipfendra for people with hypercholesterolemia, a condition marked by high levels of LDL cholesterol.
LDL is often called “bad” cholesterol because excess amounts can contribute to plaque inside arteries. The American Heart Association says about 1 in 4 U.S. adults has high LDL cholesterol, which can raise the risk of heart disease and stroke.
How the new pill works
According to the FDA and Merck, Lipfendra blocks a protein called PCSK9, which is involved in controlling LDL cholesterol levels. That separates it from statins, which act on an enzyme the liver uses to produce cholesterol.
The first PCSK9 inhibitor, Amgen’s Repatha, won approval in 2015 and is an injectable medicine. Regeneron and Sanofi also make an injectable PCSK9 treatment.
The key shift with Lipfendra is form: patients who are prescribed this type of treatment now have an oral option in a category known for injections.
Trial results showed sharp LDL drops
Merck reported in a late-stage clinical trial that Lipfendra reduced LDL cholesterol by as much as 60% after 24 weeks.
A second trial looked at people with heterozygous familial hypercholesterolemia, a genetic condition that causes very high cholesterol from birth. Researchers reported similar reductions in that group.
In both trials, nearly all participants were already using a statin, and many were also taking ezetimibe, another cholesterol-lowering drug.
Approval lands after tougher cholesterol targets
The approval comes months after several major medical groups, including the American Heart Association and the American College of Cardiology, issued updated guidance calling for more aggressive LDL control.
Under those March guidelines, people without heart disease risk factors should aim for LDL cholesterol below 100 mg/dL. People at high risk should target below 70 mg/dL, while people with heart disease should aim for below 55 mg/dL.
Merck says Lipfendra will list at $10.50 per day, or $315 for a 30-day supply. The company said it expects patients’ out-of-pocket costs to be lower.
This story draws on original reporting from NBC News.