WASHINGTON — Federal health regulators have given the green light to a first-of-its-kind oral medication capable of sharply cutting cholesterol levels — a benefit that until now has only been achievable through costly injections.
The drug, developed by Merck and approved Thursday, is intended for patients whose artery-clogging cholesterol remains dangerously high even after taking statins — the go-to medications doctors prescribe to reduce heart attack risk. The new pill will be sold under the brand name Lipfendra.
What sets Lipfendra apart is how it works: it’s the first pill to block a liver protein known as PCSK9, which normally limits the body’s ability to flush cholesterol out of the bloodstream. Injectable medications targeting this same protein have been on the market from Amgen and other pharmaceutical companies for over a decade, but widespread use has been hampered by steep costs, insurance hurdles, and doctors being slow to prescribe them.
Statins remain the foundation of cholesterol treatment by reducing how much cholesterol the liver produces. However, even at maximum doses, many patients still can’t get their LDL — commonly called “bad” cholesterol — down to the levels recommended by medical guidelines. That’s where Lipfendra is expected to fill a gap.
Merck, based in Rahway, New Jersey, earned the FDA’s approval after presenting results from two clinical studies involving high-risk patients who added the pill to their existing treatment regimens, including statins. In one trial involving 3,000 participants, patients taking Lipfendra experienced a drop in LDL cholesterol of more than 55% after six months. A second study found an average reduction of 59% compared to patients who received a placebo.
Researchers noted that the cholesterol-lowering effect held up well over a full year, declining only slightly. Side effects such as dizziness and diarrhea were reported at similar rates among both those taking Lipfendra and those on the placebo. One important note for patients: the pill must be taken on an empty stomach.
The FDA fast-tracked its review of Lipfendra through a special program designed to speed up approval of promising medications that serve a significant public need. That pathway was established under then-FDA chief Dr. Marty Makary, who stepped down from the agency in May following sustained pressure from pharmaceutical companies, patient advocates, and other outside groups.
Heart disease remains the top killer in the United States, with elevated LDL cholesterol — which causes plaque to accumulate in arteries — being one of the most significant risk factors for heart attacks and strokes. Medical guidelines consider an LDL level of 100 acceptable for otherwise healthy individuals, but recommend pushing it below 70 for those diagnosed with high cholesterol or heart disease, and even lower for patients at the highest risk.
