- Lepodisiran reduced lipoprotein(a) levels by 93.9% after six months.
- The drug targets mRNA to block the production of Lp(a).
- High Lp(a) levels increase the risk of heart attacks and strokes.
- The Phase 2 trial involved 320 participants.
- Findings were published in The New England Journal of Medicine.
Breakthrough in Cholesterol Treatment
An experimental drug, lepodisiran, has shown remarkable results in reducing lipoprotein(a), a dangerous form of cholesterol, by up to 94% in a 2025 clinical trial. Lipoprotein(a), or Lp(a), is a genetic risk factor for cardiovascular disease that has long been considered untreatable. The findings, presented at the American College of Cardiology’s annual meeting and published in The New England Journal of Medicine, mark a significant advancement in cardiovascular health.
How Lepodisiran Works
Lepodisiran targets messenger RNA (mRNA), which instructs the body to produce Lp(a). By blocking this process, the drug effectively reduces Lp(a) levels. Dr. Steven Nissen, the lead researcher of the trial, described the results as groundbreaking. “We have never been able to treat lipoprotein(a) until now,” he said. The Phase 2 trial, funded by Eli Lilly, involved 320 participants. A single injection reduced Lp(a) levels by 93.9% after six months, with effects remaining strong at 88.5% after one year.
The Dangers of Lipoprotein(a)
Lipoprotein(a) is particularly harmful because it binds to LDL cholesterol, increasing the risk of arterial blockages. It also promotes inflammation and blood clot formation. Despite its dangers, routine cholesterol tests do not typically measure Lp(a) levels, as there has been no effective treatment until now. Dr. Eric Brandt, a preventive cardiologist at the University of Michigan, emphasized the potential impact of lepodisiran. “These drugs have the potential to nearly eliminate that lipoprotein,” he said.
Patient Stories Highlight the Need
Donald Kosec, a 61-year-old from Ohio, shared his experience with high Lp(a) levels. Despite maintaining a healthy lifestyle, Kosec underwent quintuple bypass surgery after discovering severe arterial blockages. He participated in a clinical trial for a similar drug, olpasiran, but later learned he had received a placebo. “I’ll be much happier when I can get on a drug that actually improves that,” he said.
Future Implications
The success of lepodisiran opens new possibilities for treating cardiovascular disease, particularly for the estimated 64 million Americans with elevated Lp(a) levels. Further research and larger trials are needed to confirm the drug’s long-term safety and efficacy. If approved, lepodisiran could become a vital tool in preventing heart attacks, strokes, and early deaths caused by high Lp(a).