A novel oral medication, enlicitide, has demonstrated substantial efficacy in lowering “bad” cholesterol (LDL) levels in individuals with familial hypercholesterolemia, a genetic disorder causing dangerously high cholesterol from birth. Results from a year-long phase 3 clinical trial, presented at the American Heart Association meeting and published in the Journal of the American Medical Association, show a dramatic reduction in LDL compared to placebo.
Key Findings of the Trial
The study involved approximately 300 adults already on standard statin therapy, all inheriting the condition from one parent. Participants taking enlicitide daily for 52 weeks experienced, on average, a 58% drop in LDL cholesterol within the first 24 weeks. This contrasted sharply with the placebo group, which saw a nearly 3% increase in LDL during the same period. After a full year, the drug group maintained a 55% LDL reduction, while the placebo group’s levels rose almost 9%.
These findings are significant because familial hypercholesterolemia affects roughly 1 in 250 people and predisposes them to early-onset cardiovascular disease. Even with existing treatments, many patients struggle to achieve optimal cholesterol levels.
How Enlicitide Works
Enlicitide targets PCSK9, a protein responsible for breaking down LDL receptors in the liver. By inhibiting PCSK9, the drug allows more LDL receptors to remain active, boosting the liver’s ability to remove cholesterol from the bloodstream.
While injectable PCSK9 inhibitors have been available for over a decade, their high cost and administration barriers have limited widespread use. An oral alternative like enlicitide could offer a more accessible and convenient solution.
Implications for Patients
The study highlights the potential of enlicitide to address a critical unmet need in managing familial hypercholesterolemia. Lowering LDL cholesterol significantly reduces the risk of heart attack, stroke, and other cardiovascular events.
Although further research is needed, including long-term studies, the results suggest that enlicitide could become a valuable addition to the arsenal of cholesterol-lowering therapies. The drug’s oral administration also represents a major advantage over current injectable options, potentially improving patient adherence and accessibility.
In conclusion, the clinical trial data demonstrate that enlicitide effectively reduces LDL cholesterol in patients with familial hypercholesterolemia, offering a promising new treatment option for this high-risk population
