A new pill developed by researchers at Monash University in Australia has reduced a dangerous and previously untreatable form of cholesterol by up to 65% in early clinical trials — offering the first real hope for millions of people whose elevated cholesterol is driven by genetics rather than lifestyle.
At a glance
- Muvalaplin: The world’s first oral drug designed specifically to target lipoprotein(a), or Lp(a), a genetic form of “bad cholesterol” that statins and lifestyle changes cannot effectively control.
- Lp(a) reduction: Trial participants taking doses of 100 mg or more saw Lp(a) levels fall by 64% to 65% within 14 to 15 days, with measurable drops appearing as early as day two.
- Phase 1 trial: The study enrolled 114 participants — 89 receiving muvalaplin and 25 receiving a placebo — and reported no deaths or serious adverse events across either group.
Why Lp(a) has been so hard to treat
Lipoprotein(a) is a type of low-density lipoprotein — LDL, the cholesterol most people know as the “bad” kind — but it behaves differently and more dangerously. It is stickier than ordinary LDL, meaning it is more likely to build up in artery walls, trigger clots, and drive the condition known as atherosclerosis.
High Lp(a) significantly raises the risk of heart attack and stroke, and the risk is even greater in people with familial hypercholesterolemia, an inherited condition marked by persistently elevated cholesterol. Because Lp(a) is largely determined by genetics, it does not respond the way ordinary cholesterol does to diet, exercise, or commonly prescribed statins.
Until now, there were no approved medications that specifically targeted it. As lead researcher Stephen Nicholls put it: “Lp(a) is essentially a silent killer with no available treatment.”
How muvalaplin works
Lp(a) is formed when two molecules bind together: apolipoprotein B100 (apo B100), found in LDL particles, and a glycoprotein called apolipoprotein(a), or apo(a). Muvalaplin works by interrupting that binding process, preventing the body from assembling Lp(a) in the first place.
The trial ran in two phases. The first gave single doses — ranging from 1 mg to 800 mg — to healthy participants with an average age of 29. The second gave daily doses over 14 days to participants with an average age of 32 who already had elevated Lp(a) levels. The clearest results came from that second group, where doses of 100 mg or more produced reductions of 64% to 65%.
Reported side effects included headache, diarrhea, abdominal pain, nausea, and fatigue — but no serious adverse events occurred in either group.
What comes next
The researchers are clear that this is a first step. The study was small, short, and drew from a relatively narrow population with low-to-moderate Lp(a) elevation. Larger trials — involving people with established cardiovascular disease and higher baseline Lp(a) levels, and drawn from more diverse populations — will be needed before the drug can move toward approval.
Still, the significance of what has been shown is hard to overstate. For a condition that has effectively had no pharmacological solution, a 65% reduction in a two-week trial is a meaningful signal. And the fact that muvalaplin is a pill, not an injection, matters enormously for access. “Being able to deliver it in an oral tablet means it will be more accessible for patients,” Nicholls said.
The study was published in JAMA Network and builds on a growing body of cardiovascular research coming out of Monash University’s cardiology group. Globally, an estimated one in five people have elevated Lp(a) — a population that has long had nowhere to turn for targeted treatment. Broader cardiovascular research, including work from the American Heart Association, has consistently flagged Lp(a) as an underappreciated risk factor.
Roughly 1.4 billion people worldwide have some form of high cholesterol, and the subset driven by genetic factors has remained frustratingly out of reach. If muvalaplin clears larger trials, it could change the outlook for tens of millions of patients who have been told, in effect, that their risk is baked into their DNA and there is nothing to be done about it.
Read more
For more on this story, see: New Atlas
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