FDA Approves First Oral PCSK9 Inhibitor for High Cholesterol

The FDA approved Merck's Lipfendra (enlicitide) on Friday, the first oral PCSK9 inhibitor for adults with high cholesterol. The once-daily pill provides an alternative for patients who previously had to use injectable therapies, which require shots every two to four weeks.

PCSK9 inhibitors lower LDL cholesterol by blocking a protein that interferes with the liver's ability to remove the particle from the blood. Until now, all approved drugs in the class were injectable. The oral formulation is intended for adults who need further LDL reduction despite existing treatments, including statins and injectable PCSK9 drugs, the agency said.

"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," said Michael Davis, M.D., Ph.D., Acting Director of the FDA's Center for Drug Evaluation and Research. "This approval provides an additional treatment option for adults with hypercholesterolemia and reflects the FDA's broader commitment to supporting meaningful advances in patient access and tackling chronic diseases."

High cholesterol affects roughly one in three U.S. adults. Excess LDL-C accumulates in artery walls over time, forming plaques that narrow blood vessels and raise the risk of heart attack or stroke. Because the condition causes no symptoms, many patients are diagnosed only through routine blood testing. Diet, physical inactivity, excess body weight, and inherited conditions all contribute to elevated levels.