FDA approves a first-of-its-kind pill to cut cholesterol in high-risk patients

The Food and Drug Administration has approved a first-of-its-kind pill that can drastically reduce cholesterol in a way that’s previously only been available with expensive, injectable drugs.

The drug from Merck was OK’d on Thursday for patients with artery-clogging cholesterol that persists even after taking statins, the standard medications for cutting heart attack risk. Merck will market its pill under the brand name Lipfendra.

It’s the first noninjectable medication that works by blocking a liver protein called PCSK9. That protein limits the body’s ability to clear cholesterol from the blood, and biotech injectables targeting it have been available from Amgen and other drugmakers for more than a decade. But patient access has been stymied for years by high prices, insurance restrictions and limited prescribing by doctors.

Statins block some of the liver’s production of cholesterol and are the cornerstone of treatment. But even at the highest doses, many people need additional help lowering their LDL, or bad, cholesterol enough to meet medical guidelines.

Merck, which has headquarters in Rahway, New Jersey, won approval based on two studies in high-risk patients who added the company’s pill to their standard treatment, including statins. In one study of 3,000 patients, those taking Lipfendra saw their levels of LDL cholesterol drop more than 55% after six months. In a second study, patients averaged a reduction of 59% compared with patients who received a dummy pill.

That benefit dropped only slightly over a year, and side effects — including dizziness and diarrhea — were similar between those taking the pill or a placebo, researchers found. One caveat: The pill must be taken on an empty stomach.

The FDA reviewed the drug under its program that promises ultra-fast reviews for promising medications that serve the public interest. The pathway was created by then-FDA chief Dr. Marty Makary, who resigned from the agency in May after months of pressure from drugmakers, patients and other outside groups.

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.