FDA clears Merck’s daily PCSK9 cholesterol pill
Lipfendra is the first oral PCSK9 inhibitor approved to lower LDL cholesterol, a drug class previously sold as injections.
By Tom Brennan · Health & Medicine Correspondent
2 min read
The Food and Drug Administration approved Merck’s Lipfendra on Thursday, making it the first pill in a powerful class of cholesterol medicines that has been available only by injection. The approval gives patients with high LDL cholesterol another option beyond statins and injectable PCSK9 drugs.
The FDA said Lipfendra is approved for people with hypercholesterolemia, a condition marked by elevated LDL cholesterol. LDL is often described as “bad” cholesterol because excess levels can contribute to artery-clogging plaque, according to the American Heart Association.
About one-quarter of U.S. adults have high LDL cholesterol, the American Heart Association said. The group says high LDL can raise the risk of heart disease and stroke by promoting plaque buildup in the arteries.
How the drug works
Lipfendra is a PCSK9 inhibitor, according to the FDA. The medicine blocks a protein involved in cholesterol regulation, which helps reduce LDL levels.
Statins lower cholesterol through a different mechanism, targeting an enzyme the liver uses to produce cholesterol. In the clinical studies cited for Lipfendra, nearly all participants were already taking a statin, and many were also taking ezetimibe, another cholesterol-lowering medicine.
Merck reported that Lipfendra reduced LDL cholesterol by as much as 60% after 24 weeks in a late-stage clinical trial. A separate trial involving people with heterozygous familial hypercholesterolemia, a genetic condition that causes very high cholesterol from birth, showed comparable reductions, according to the published study results.
A pill in a drug class built on injections
The first PCSK9 inhibitor, Amgen’s Repatha, won approval in 2015, according to the FDA history cited for the drug class. Regeneron and Sanofi also sell an injectable PCSK9 inhibitor, Praluent.
Lipfendra changes the form of treatment in that class from a shot to a once-daily tablet. The approval does not make it a replacement for every cholesterol medicine; the trials described Lipfendra as being used in patients who were often already receiving other LDL-lowering therapy.
The approval comes after major medical groups called for tighter cholesterol control. In March, organizations including the American Heart Association and the American College of Cardiology issued guidelines recommending LDL targets below 100 mg/dL for people without heart disease risk factors, below 70 mg/dL for high-risk patients and below 55 mg/dL for people with heart disease.
Merck said Lipfendra will have a list cost of $10.50 per day, equal to $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.