A new drug called muvalaplin successfully lowers a genetic form of cholesterol that’s linked to higher risks of strokes and heart attacks, according to a new study published Monday in JAMA. It’s the first treatment for this form of cholesterol.

The drug targets lipoprotein(a), or Lp(a), a mostly genetic form of cholesterol that raises the risk of heart attack and stroke. Having too much Lp(a) — also called “LP little A” — affects about 1 in 5 people worldwide. Muvalaplin stops the body from letting Lp(a) form. In the study, it lowered levels of Lp(a) by as much as 65%.

Currently, there’s no approved treatment on the market. 

Lp(a) isn’t the same as another type of cholesterol called low-density lipoprotein, or LDL, known as the “bad cholesterol.” Lp(a) is more sticky, so having it raises the risk for blockages and blood clots. Statins, which are typically prescribed for high cholesterol, can regulate LDL levels, but they don’t have the same effect on Lp(a). Instead, Lp(a) is hard to control with diet, exercise and lifestyle modifications. It’s more genetically influenced. 

Researchers discovered Lp(a) more than 50 years ago, but there haven’t been any treatments to lower levels of Lp(a) until now. They struggled to find a working format when they did have a drug that helped. Muvalaplin is now in pill form.

Stephen Nicholls, MD, a cardiologist and director of the Monash University’s Victorian Heart Institute and Victorian Heart Hospital, led the study. The research was recently presented at the European Society of Cardiology Congress in Amsterdam.

“When it comes to treating high Lp(a), a known risk factor for cardiovascular disease, our clinicians currently have no effective tools in their kit,” Nicholls said. “This drug is a game-changer in more ways than one. Not only do we have an option for lowering an elusive form of cholesterol, but being able to deliver it in an oral tablet means it will be more accessible for patients.”

“Lp(a) is essentially a silent killer with no available treatment, this drug changes that,” he added.

This successful trial means that muvalaplin now will go on to larger clinical trials.