New oral drug shows promising results in reducing LDL cholesterol

A recent medical study has demonstrated remarkable results for an oral medication that effectively reduces bad cholesterol (LDL) levels by nearly 60% in patients with heterozygous familial hypercholesterolemia (HeFH).

The research, published in the Journal of the American Medical Association (JAMA) and highlighted by MedicalXpress, evaluated the experimental drug Enlicitide, developed by the U.S.-based company Merck.

Enlicitide is part of the advanced class of PCSK9 inhibitors, which traditionally are administered via injections. For the first time, the treatment has been formulated as a pill, offering a more convenient alternative to injections.

Heterozygous familial hypercholesterolemia is a genetic condition caused by a mutation in a single gene, affecting approximately 1 in 250 people.

This mutation impairs the body’s ability to remove LDL cholesterol from the bloodstream, leading to its accumulation in arteries, which increases the risk of heart disease and stroke.

Although statins and other cholesterol-lowering medications exist, many patients—especially those with hereditary forms of the disease—struggle to achieve target LDL levels.

Enlicitide works by binding to PCSK9 proteins in the blood, preventing them from breaking down liver receptors responsible for clearing LDL cholesterol.

By protecting these receptors, the drug enhances their activity, allowing the liver to remove more LDL cholesterol from the bloodstream and significantly lowering overall levels.

This oral formulation could mark a major advancement in the treatment of hereditary high cholesterol, offering patients an effective, needle-free alternative.