Experimental pill may offer new cholesterol-lowering option for millions

There are other pills that patients can add to their statin

An illustration of cholesterol formation, fat, artery, vein, heart.

A novel experimental pill has shown significant promise in sharply reducing artery-clogging cholesterol among individuals who remain at high risk of heart attacks, despite already being on statin medication. Researchers unveiled these findings on Wednesday, highlighting a potential breakthrough in cardiovascular care.

The pill, named enlicitide, operates by helping the body eliminate cholesterol, a mechanism currently only achievable through injected medicines. Should it gain approval from the US Food and Drug Administration, enlicitide could offer a more convenient, oral treatment option for millions of patients worldwide.

While statins are the established cornerstone of treatment, working to block the liver's cholesterol production, many individuals find that even the highest doses are insufficient to lower their "bad" LDL cholesterol to medically recommended levels. This new oral therapy aims to address that critical unmet need.

In a major study, more than 2,900 high-risk patients were randomly assigned to add a daily enlicitide pill or a dummy drug to their standard treatment. The enlicitide users saw their LDL cholesterol drop by as much as 60% over six months, researchers reported in the New England Journal of Medicine.

There are other pills that patients can add to their statins “but none come close to the degree of LDL cholesterol lowering that we see with enlicitide,” said study lead author Dr. Ann Marie Navar, a cardiologist at UT Southwestern Medical Center.

That benefit dropped only slightly over a year, and there was no safety difference between those taking the pill or placebo, researchers found. One caveat: The pill must be taken on an empty stomach.

A monitor shows an artery during a catheterization lab heart procedure in Burlington, Vt., Tuesday, Sept. 23, 2008. (AP Photo/Toby Talbot, File) (Copyright 2026 The Associated Press. All rights reserved.)

Heart disease is the nation’s leading cause of death and high LDL cholesterol, which causes plaque to build up in arteries, is a top risk factor for heart attacks and strokes. While an LDL level of 100 is considered fine for healthy people, doctors recommend lowering it to at least 70 once people develop high cholesterol or heart disease — and even lower for those at very high risk.

Statin pills like Lipitor and Crestor, or their cheap generic equivalents, are highly effective at lowering LDL. For additional help, some powerful injected drugs work differently, blocking a liver protein named PCSK9 that limits the body’s ability to clear cholesterol from blood. Yet only a small fraction of people who could benefit from PCSK9 inhibitors use them. While prices for the costly shots have dropped recently, patients still may dislike administering shots and Navar said they’re more complex for doctors to prescribe.

Merck funded Wednesday’s study, which provides some of the final data needed to seek FDA approval of enlicitide. The FDA has added the drug to a program promising ultra-fast reviews.

The research offers “compelling evidence” that the new pill lowers cholesterol about as much as those PCSK9 shots, Dr. William Boden of Boston University and the VA New England Healthcare System, who wasn’t involved with the study, wrote in the journal.

Boden cautioned there’s no data yet showing the pill’s cholesterol-reduction translates into fewer heart attacks, strokes and death. That takes much longer than a year to prove. Merck has a study of more than 14,000 patients underway to tell.