A new drug, lepodisiran, dramatically reduces lipoprotein(a), a major genetic driver of heart attacks and strokes, with effects lasting nearly a year from just one dose.
- A single dose of lepodisiran reduced lipoprotein(a) by 94% for six months
- Lipoprotein(a) is a genetic risk factor for heart attack and stroke unaffected by diet or exercise
- The therapy showed no major safety concerns in the Phase 2 trial involving 320 patients
A single dose of an experimental therapy called lepodisiran safely lowered average blood levels of lipoprotein(a) by 94% over 180 days (1✔ ✔Trusted Source
Lepodisiran – A Long-Duration Small Interfering RNA Targeting Lipoprotein(a)
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).
After a full year, levels remained reduced by 88.5%. Lipoprotein(a) is a major contributor to heart disease and stroke and has historically been seen as an untreatable risk factor.
These results were published in the New England Journal of Medicine.
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A single injection of #Lepodisiran has been shown to keep levels of genetically inherited cholesterol nearly undetectable for over half a year.
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Lipoprotein(a) Impact on Heart Attack and Stroke Risk
Lipoprotein(a), or Lp(a), is made in the liver and is similar to “bad” LDL cholesterol. However, Lp(a) levels are mostly (80-90%) determined by a person’s genes.
The unique structure of Lp(a) promotes plaque buildup in arteries and increases clotting, raising the risk of heart attack and stroke.
While effective drugs exist for lowering LDL cholesterol, there are currently no approved treatments to lower Lp(a). Because it is genetic, diet and exercise have little impact on its levels (2✔ ✔Trusted Source
Lepodisiran: From genetic targeting to cardiovascular promise: A detailed narrative review of the literature
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Single Dose Lepodisiran Effectiveness
The Phase 2 trial enrolled 320 patients across multiple countries with an average age of 62. Results demonstrated that a single 400mg dose of lepodisiran reduced Lp(a) by 93.9% from day 60 to 180, with no major safety issues identified.
The reduction from days 30 to 360 was 88.5% after one dose and 94.8% after two doses given 180 days apart. Lp(a) levels stayed 53.4% below baseline 540 days after a single dose and 74.2% lower 360 days after a second dose.
Mild Injection Site Reactions Reported
No major safety concerns arose during the trial. Up to 10% of patients experienced mild, short-lived injection site reactions such as pain or redness.
Several therapies like lepodisiran, which work by “silencing” the gene that produces a key part of Lp(a), are in development. High Lp(a) is estimated to affect 64 million people in the United States and 1.4 billion globally.
Importance of Blood Testing for High-Risk Patients
“Many people with high Lp(a) don’t experience symptoms, and unfortunately, it is not frequently tested,” said lead author Dr. Steven Nissen of Cleveland Clinic. He advises patients with a personal or family history of heart disease to ask their doctor for an Lp(a) blood test.
To sum up, the experimental drug lepodisiran represents a significant breakthrough, offering a potent, safe, and long-lasting reduction of lipoprotein(a), a stubborn genetic risk factor for cardiovascular disease, moving us closer to an effective treatment for millions of people.
Frequently Asked Questions
Q: What is lepodisiran used for?
A: It is designed to lower lipoprotein(a), a genetic risk factor for heart disease and stroke.
Q: Was the treatment safe?
A: No major safety concerns were reported, with only mild injection site reactions in some patients.
Q: How long do the effects of a single dose last?
A: A single injection reduced lipoprotein(a) levels by an average of 94% for 180 days, with effects measurable a full year later.
Q: Who should get tested for high lipoprotein(a)?
A: Individuals with a personal or family history of heart disease should speak to their doctor about getting a simple blood test for Lp(a).
References:
- Lepodisiran – A Long-Duration Small Interfering RNA Targeting Lipoprotein(a) – (https://www.nejm.org/doi/full/10.1056/NEJMoa2415818)
- Lepodisiran: From genetic targeting to cardiovascular promise: A detailed narrative review of the literature – (https://pmc.ncbi.nlm.nih.gov/articles/PMC12426975/)
Source-Medindia