For the first time, doctors may soon have a way to lower extremely high levels of a risky type of cholesterol known as lipoprotein(a), or Lp(a). This genetically-influenced cholesterol has long been untreatable, leaving many patients at heightened risk of heart disease. But thanks to a new breakthrough drug called muvalaplin, there is now hope for better controlling this “silent killer.”

Lp(a) is a form of LDL or “bad” cholesterol. But unlike regular LDL, it’s stickier and more likely to build up in arteries. This can lead to blood clots, heart attacks, strokes and other cardiovascular disasters.

To make matters worse, high Lp(a) is largely inherited. So healthy habits like diet, exercise and quitting smoking don’t do much to lower levels. Even cholesterol medicines like statins have little effect on this stubborn form of LDL.

That’s why the recent development of muvalaplin marks such an exciting medical advance.

In a pioneering new study, researchers found that muvalaplin significantly lowers Lp(a) when taken as a once-daily oral tablet. The study was led by Dr. Stephen Nicholls and colleagues at Monash University in Australia.

They recruited 114 participants with elevated Lp(a) levels for a Phase 1 trial. Some received a single dose of muvalaplin up to 800 mg. Others took it daily for 14 days, again up to 800 mg per day.

The results revealed just how powerful muvalaplin’s Lp(a)-lowering effects are. With a single dose of 100 mg or more, Lp(a) levels plunged by 64-65%. And taking it daily had even more dramatic impacts.

Muvalaplin is the first oral medication specifically designed to target Lp(a). It works by interfering with the interaction between two components that make up Lp(a). This disrupts the body’s ability to assemble this harmful cholesterol.

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

The most common side effects were headache, diarrhea and abdominal pain. More testing is still needed to confirm muvalaplin’s long-term safety. But this early research demonstrates the exciting potential of muvalaplin to mitigate cardiovascular risk.

For those genetically prone to extremely high Lp(a), this drug may be life-altering. Conditions like familial hypercholesterolemia often involve elevated Lp(a). This “silent killer” cholesterol silently boosts the likelihood of atherosclerosis, blood clots and arterial blockages.

By specifically targeting Lp(a), muvalaplin provides fresh hope of preventing heart attacks and strokes in those patients most in need. It works in an entirely different manner than statins, niacin, ezetimibe or other current options.

As Dr. Nicholls aptly stated “this drug is a game-changer in more ways than one.” Not only can it lower a tricky form of cholesterol, but its once-daily oral delivery makes it far more accessible.

While more testing is warranted, muvalaplin may soon offer clinicians a powerful new weapon against cardiovascular disease by silencing this “silent killer.” For many high-risk patients, this innovative cholesterol pill could ultimately prove lifesaving.

The study was published in the journal JAMA Network.