A new daily pill designed to sharply cut levels of low-density lipoprotein cholesterol (LDL-C)—the “bad” cholesterol associated with heart attacks and strokes—is generating international interest following impressive late-stage trial results.
The experimental therapy, known as enlicitide, reduced LDL-C by 58.2% in adults with a genetic cholesterol disorder, according to findings published in the Journal of the American Medical Association (JAMA).
Its potential to deliver substantial cholesterol lowering through a simple oral dose, instead of regular injections, has sparked strong enthusiasm among heart specialists looking for easier, patient-friendly treatment options.
The phase 3 trial assessed 303 adults diagnosed with heterozygous familial hypercholesterolaemia (HeFH)—a hereditary condition that keeps LDL levels abnormally high and increases the risk of early cardiovascular disease. The study took place across 59 medical centres in 17 countries.
Significance for India
With cardiovascular disease remaining a major cause of death in India, a potent and easy-to-use cholesterol-lowering pill could have far-reaching benefits.
Recent data underscore India’s severe lipid burden. A nationwide survey conducted by major research institutions and published in The Lancet in 2023 revealed that over 81% of Indians have abnormal lipid levels, while 20.9%—approximately 185 million people—show elevated LDL-C.
To address this concern, India’s first dedicated lipid management guidelines issued in July 2024 advise individuals at high cardiovascular risk, including those with diabetes or hypertension, to keep LDL-C levels below 70 mg/dL.
Yet despite the widespread use of statins, which are the primary therapy for high cholesterol, about one-fifth of patients still fail to reach recommended LDL targets. Some also experience side effects such as muscle pain, leaving them unable to tolerate long-term statin therapy. For these groups, alternative treatments remain essential.
How the New Pill Functions
Enlicitide works within the same therapeutic class as several injectable PCSK9 inhibitors already approved in India. These medicines target PCSK9, a protein that limits the liver’s ability to filter LDL cholesterol from the blood.
By switching off the gene that triggers PCSK9 production, enlicitide helps the liver clear LDL-C more effectively, resulting in significantly lower cholesterol levels.
What’s Next
Although the trial results are encouraging, the study focused only on LDL-C reduction. It did not measure whether the drug decreases the risk of heart attacks, strokes, or cardiovascular-related deaths—crucial data required before broader clinical adoption.
Long-term safety findings are also awaited, particularly important for a treatment intended for continuous use.