When the FDA approved bempedoic acid, marketed under the brand name Nexletol, back in 2020, it was clear that the drug helped lower LDL — "bad" cholesterol. The drug was intended for people who can't tolerate statin medications due to muscle pain, which is a side effect reported by up to 29% of people who take statins.

What was unknown until now, is whether bempedoic acid also reduced the risk of cardiovascular events. Now, the results of a randomized, controlled trial published in The New England Journal of Medicine point to significant benefit. The study included about 14,000 people, all of whom were statin intolerant.

"The big effect was on heart attacks," says study author Dr. Steven Nissen of Cleveland Clinic.

People who took daily doses of bempedoic acid for more than three years had about a 23% lower risk of having a heart attack, in that period, compared to those taking a placebo. There was also a 19% reduction in coronary revascularizations, which are procedures that restore blood flow to the heart, such as a bypass operation or stenting to open arteries.

With these findings, the benefits of the medication are now clearer, says Dr. John Alexander, a cardiologist and professor at Duke University. "Bempedoic acid has now entered the list of evidence-based alternatives to statins," Alexander wrote in an editorial, published alongside the study.

Jennifer Kluczynski, 55, of Lambertville, Mich., had tried multiple statins but experienced lots of muscle aches and pains. "I felt like I had the flu" without the fever, she explains. Some days she just wanted to go back to bed. Her doctor prescribed Nexletol about two years ago, and she says she feels much better and hasn't "been achy."

And her cholesterol levels remain well controlled by the medicine.

"This is working for me wonderfully and I'm not having any side effects," Kluczynski says.

Bempedoic acid is a prodrug, which means it is activated by an enzyme after the medication enters the body. And, unlike statin drugs, bempedoic acid is mostly metabolized in the liver, not in peripheral tissues, like muscle, so Alexander says it "has few, if any, muscle-related side effects." In the clinical trial, myalgias, which are muscle aches or pains, were reported more among people taking the placebo (6.8%), compared to those taking bempedoic acid (5.6%).

Researchers say bempedoic acid was generally well-tolerated by people in the trial but there were some reported risks, including an increased incidence of gout, which was reported in 3% of the bemepedoic acid group, compared to 2% of the placebo group. And the study also found a small increase in the number of people who developed gallstones (2% in the bempedoic group, 1% in the placebo group). But the benefits of taking the drug " far outweigh the small risks that we observed in the trial," study author Nissen told NPR.

The study was funded in part by the maker of the drug, Esperion Therapeutics, but Nissen explains his team works independently. "My statisticians generated all the numbers in the manuscript," he says. "We do our own analyses and we report the adverse events very carefully because every drug has benefits and risks."

It's important to point out that statins are very well-tolerated by millions of people, Nissen says, and there's "enormous amounts of evidence that they reduce the risk of heart attack, stroke and death from cardiovascular causes."

Statins are also relatively inexpensive with many patients paying less than $10 a month, given the many options, including generics. Kluczynski's insurance plan covers the cost of Nexletol, but it can cost about $400 per month for people who are not covered by insurance. There is currently no generic for Nexletol.

Nissen says statins will "continue to be the cornerstone of therapy to prevent cardiovascular events." But for people who simply cannot tolerate a statin, he says, "we have an alternative for them."