A new oral medication, enlicitide decanoate, has demonstrated a significant reduction in low-density lipoprotein cholesterol (LDL-C) – commonly known as “bad” cholesterol – in a recent clinical trial. The drug lowered LDL-C levels by an average of 58.2% in patients with a genetic condition that causes dangerously high cholesterol, offering a promising new treatment option for those who don’t respond well to existing medications.
The Challenge of High Cholesterol
High LDL-C is a major risk factor for heart disease and stroke, affecting tens of millions worldwide. The buildup of cholesterol in arteries restricts blood flow, increasing the likelihood of heart attacks and strokes over time. For individuals with heterozygous familial hypercholesterolemia (HeFH) – a genetic disorder affecting roughly 1 in 250 people – managing cholesterol is particularly critical, as elevated levels begin at birth.
Trial Results: A Pill That Works
Researchers conducted a randomized, placebo-controlled trial involving 293 participants with HeFH already taking statins that weren’t adequately controlling their cholesterol. The results were clear: after 24 weeks, those who received the once-daily enlicitide decanoate pill experienced a substantial drop in LDL-C. In contrast, the placebo group saw a slight increase in cholesterol levels.
Notably, the drug remained effective over a longer 52-week period, with consistent biomarker improvements and few reported side effects. Importantly, 98% of participants adhered to the medication regimen, suggesting high tolerability and ease of use.
How Enlicitide Works
Enlicitide decanoate is a PCSK9 inhibitor. This means it boosts the liver’s ability to clear LDL-C from the bloodstream, essentially removing an obstacle in the body’s natural cholesterol-regulation process. Existing PCSK9 inhibitors require injections, which can deter some patients. This new pill form may improve treatment compliance.
What’s Next?
While the trial results are encouraging, enlicitide decanoate still requires regulatory approval before becoming widely available. Future research will focus on whether the reduction in LDL-C translates to fewer heart attacks and strokes in the long run, as well as broader testing across a wider patient population.
The study reinforces the importance of proactive cholesterol management, particularly for individuals with genetic predispositions. Left untreated, elevated LDL-C leads to premature cardiovascular disease due to prolonged exposure.
