WASHINGTON – The FDA on Friday approved the first non-statin oral medication for high cholesterol in almost two decades, giving patients and doctors another weapon against heart disease, the number one killer in the United States and around the world.
The drug, bempedoic acid, is the first in a new class of drugs to treat low-density-lipoprotein cholesterol, also called “bad” cholesterol, which causes the buildup of fatty deposits in the arteries, reducing blood and oxygen flow.
Cardiologists said the new drug, which is taken as a pill once a day, will be used primarily as an add-on therapy for people who are taking as high a dose of statin medications as they can tolerate but still have higher-than-desired cholesterol. Studies showed that bempedoic acid reduced cholesterol by an average of 18 percent in patients taking moderate- or high-dose statins, compared to the placebo group, said the manufacturer, Esperion Therapeutics Inc.
The medicine also can be used for patients who can tolerate only low-dose statins or can’t take statins at all. In studies involving those patients, it reduced LDL cholesterol by 28 percent, the company said.
Esperion, which is based in Ann Arbor, Michigan, said the drug’s brand name will be Nexletol. Esperion also has applied to the FDA for approval of a combination pill of bempedoic acid and ezetimibe, a non-statin pill approved in 2002. In studies, the combination pill reduced cholesterol by about 38 to 44 percent, depending on whether a person was taking a statin and how much, the company said. The FDA decision is expected shortly.
Statins, which were introduced in the 1980s, are the premier medicines for high cholesterol and typically are the first drugs prescribed along with changes in diet and exercise. But they can cause muscle aches and cramps, making it hard for some people to take the required dose, said Robert Rosenson, director of cardiometabolic disorders for Mount Sinai Hospital. More than 10 percent of people taking high-dose statins had muscle pain, according to a 2005 study.
Because bempedoic acid targets an enzyme in the liver, not in muscles, it doesn’t cause muscle pain, he said.
Rosenson, who wasn’t involved in the development of bempedoic acid, said the drug reduces cholesterol by a relatively modest amount but could nevertheless be helpful in achieving patients’ goals.
Recommended cholesterol levels vary by age and gender, but the “optimal” level for LDL cholesterol is 100 milligrams per deciliter or below, according to the National Heart, Lung and Blood Institute. Experts say high-risk individuals, including those who have had a heart attack, should aim for below 70 mg/dl.
The FDA approved bempedoic acid based on its cholesterol-lowering effect. Whether the drug reduces the risk of heart attacks and strokes is being studied as part of a large outcomes trial involving people who can’t tolerate statins. The trial is scheduled to be completed in two years.
Bempedoic acid also can be used with another class of heart drugs, called PCSK9 inhibitors, which are injectable medications launched in 2015. They can reduce LDL cholesterol sharply when combined with a statin. But they initially cost about $14,000 a year, causing insurers to balk.
Since then, prices have fallen sharply, but uptake remains slow. While the drugs “are highly effective, there are a lot of hassles with prior authorization and they often get denied,” said Christie Ballantyne, a cardiologist at Baylor College of Medicine who ran the late-stage clinical trial for bempedoic acid.
Ballantyne said that bempedoic acid, along with the possible new combination with ezetimibe, will give doctors and patients more ways to attack high cholesterol – an approach that has worked to treat high blood pressure. “For blood pressure, we tend to use lots and lots of combinations, often in the same tablet, and have been pretty successful,” he said.
The Food and Drug Administration approved the drug for patients with heart disease who need to reduce their cholesterol as well as for those who with an inherited condition that causes high cholesterol levels. But doctors said they expected the drug to be prescribed more broadly – though that will depend partly on the price and insurers’ reactions.
Esperion officials did not announce a price on Friday but previously indicated it would be about $10 a day, which would make the drug less expensive than the PCSK9 inhibitors but more costly than statins, which are available in cheap generic versions.