Breakthrough Drug Lepodisiran Slashes Heart Attack and Stroke Risk by Targeting “Hidden” Cholesterol

Illustration of Lepodisiran reducing cholesterol buildup in arteries

A new drug is making waves in the fight against heart disease and stroke—and it doesn’t target the usual suspects like LDL or triglycerides. Instead, it goes after a stealthier risk factor: lipoprotein(a), or Lp(a), a lesser-known cholesterol particle that’s genetically inherited and notoriously hard to treat.

Researchers at UVA Health, in collaboration with pharmaceutical giant Eli Lilly, are testing Lepodisiran, an experimental therapy that may finally offer a solution for the estimated one in four people with dangerously high Lp(a) levels.


What Makes Lipoprotein(a) So Dangerous?

While most people are familiar with “bad cholesterol” (LDL), Lp(a) is different. It’s a sticky, genetically determined cholesterol particle that contributes to plaque buildup in arteries. Elevated Lp(a) levels can increase the risk of heart attack and stroke—regardless of how healthy your lifestyle is.

Even worse? Standard cholesterol-lowering drugs like statins don’t work on Lp(a). Until now, there’s been no approved treatment specifically targeting this hidden threat.


Lepodisiran: A Game-Changer in Cardiovascular Medicine?

In a Phase 2 clinical trial, Lepodisiran reduced Lp(a) levels by an astonishing 95% in participants receiving the highest dose. And that was with just two injections, four months apart. Even six months after a single injection, levels remained dramatically lower than baseline.

  • Participants: Adults over 40 with extremely high Lp(a)
  • Dosage: Two injections four months apart
  • Result: 95% average reduction in Lp(a) levels
  • Side effects: Mostly minor, such as injection site reactions

Dr. Christopher Kramer, Chief of Cardiovascular Medicine at UVA Health, called the results “truly promising,” emphasizing the potential of Lepodisiran to fill a massive treatment gap for millions worldwide.


Why This Matters for You

Heart disease remains the #1 killer globally, and many people with healthy diets and regular exercise still suffer unexpected cardiovascular events. Genetics play a massive role, and Lp(a) is a prime example.

If Lepodisiran proves effective in upcoming Phase 3 trials, it could revolutionize how we screen and prevent heart attacks and strokes—especially for those with family histories of early heart disease.


What’s Next?

A large-scale Phase 3 trial is now underway to determine if lowering Lp(a) actually leads to fewer heart attacks, strokes, and deaths. If successful, Lepodisiran could be on track for FDA approval and widespread clinical use.

In the meantime, if you or a loved one has a family history of heart disease or has experienced cardiac issues despite a healthy lifestyle, ask your doctor about Lp(a) testing. Awareness is the first step toward prevention.


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Article derived from: Mohammed, Z. (2025, April 8). Q&A: Could a New Drug Help Prevent Heart Attacks and Strokes? UVA Today. https://news.virginia.edu/content/qa-could-new-drug-help-prevent-heart-attacks-and-strokes

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