A daily pill can now do what only injections could — lower dangerously high cholesterol — after the FDA approved Lipfendra (enlicitide), the first oral PCSK9 inhibitor, on Thursday.

Cardiovascular disease remains the leading cause of death in the U.S., and high LDL cholesterol is a key driver, said Michael Davis, acting director of the FDA’s drug review center. “This approval provides an additional treatment option,” he said, noting the agency’s focus on expanding patient access.

High cholesterol affects tens of millions of Americans and is a leading driver of heart attacks and strokes. The condition typically produces no symptoms, leaving many unaware of their risk until routine bloodwork reveals it. Existing treatments — statins, ezetimibe, and injectable PCSK9 inhibitors — can lower LDL-C substantially, but the injectable options require self-injections that some patients find burdensome. Lipfendra offers the same target with a once-daily pill.

In two clinical trials spanning more than 3,200 adults on maximum statin therapy, Lipfendra cut LDL-C by roughly half. Patients with established cardiovascular disease or high risk for it saw a 56% reduction at 24 weeks, while those with familial hypercholesterolemia — a genetic form of high cholesterol — saw a 59% reduction. Both results were measured against placebo.

Side effects were generally mild. Diarrhea and dizziness appeared more often with Lipfendra than placebo in the familial hypercholesterolemia trial, while the other trial found no meaningful difference in adverse reactions between groups.

The FDA granted Priority Review and fast-tracked the application through its Commissioner’s National Priority Voucher program. Merck Sharp & Dohme holds the approval.

Source: FDA Press Release