WASHINGTON, D.C. — The U.S. Food and Drug Administration approved Merck’s Lipfendra as the first oral PCSK9 inhibitor for adults with high cholesterol, introducing a once-daily alternative to injectable drugs used when existing therapies do not reduce LDL cholesterol enough.
Lipfendra, also known as enlicitide, is approved alongside diet and exercise for adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia, an inherited condition associated with elevated low-density lipoprotein cholesterol.
The treatment is intended for patients who require additional LDL reduction despite other therapies. Existing options include statins, ezetimibe and injectable PCSK9 inhibitors.
“Cardiovascular disease remains the leading cause of death in the United States, and elevated LDL cholesterol is one of its most important modifiable risk factors,” Michael Davis, acting director of the FDA’s Center for Drug Evaluation and Research, stated.
The FDA based its approval on two randomized, double-blind, placebo-controlled trials involving 3,207 adults receiving the maximum statin dose they could tolerate.
In the first study, which enrolled patients with established atherosclerotic cardiovascular disease or a high risk of developing it, Lipfendra produced an average 56% reduction in LDL cholesterol at 24 weeks compared with placebo. Participants began the trial with a mean LDL level of 96 milligrams per deciliter.
The second trial involved adults with heterozygous familial hypercholesterolemia and a mean baseline LDL level of 119 milligrams per deciliter. Lipfendra reduced LDL cholesterol by an average of 59% at 24 weeks compared with placebo.
Adverse reaction rates in the first trial were similar between the Lipfendra and placebo groups. Diarrhea and dizziness occurred more frequently among Lipfendra recipients in the second study.
Discontinuation rates related to adverse reactions were comparable between the treatment and placebo groups across both trials.
PCSK9 inhibitors lower LDL cholesterol by blocking a protein that reduces the liver’s ability to remove cholesterol from the bloodstream. Until Lipfendra’s approval, drugs in the class were administered by injection.
High LDL cholesterol can contribute to plaque accumulation in arteries, increasing the risk of heart attack and stroke. The condition typically causes no symptoms and is often detected through routine blood testing.
The FDA granted Lipfendra Priority Review and evaluated the application through its Commissioner’s National Priority Voucher pilot program, which is designed to accelerate reviews of treatments addressing public health priorities.
The approval was granted to Merck Sharp & Dohme LLC.
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