For many people living with stubbornly high cholesterol, lifestyle changes and statin therapy can only go so far.
A recent study published in JAMA offers encouraging early evidence that a new oral drug, enlicitide, could help patients whose LDL (“bad”) cholesterol remains elevated despite standard treatment.
The experimental medication targets the same biological pathway as current PCSK9 inhibitors but, unlike those therapies, it is taken as a pill.
Researchers focused their trial on individuals with heterozygous familial hypercholesterolemia, a genetic condition that keeps LDL levels unusually high throughout life.
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This group often sees limited benefit from traditional approaches, making new treatment options especially important.
How the drug works and why it matters
PCSK9 inhibitors have changed cholesterol management for people at high cardiovascular risk, but all approved therapies in this class are injectable.
They work by preventing PCSK9 proteins from interfering with the liver’s ability to clear LDL cholesterol from the bloodstream.
Enlicitide uses a similar mechanism but is formulated as an oral medication, something many clinicians believe could greatly improve adherence.
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Physicians who were not involved in the study told Medical News Today that patients who avoid injections are typically more consistent with daily pills.
Study findings show large reductions in LDL
The year-long trial followed 303 participants across multiple countries, all of whom continued their statin therapy.
Two-thirds received enlicitide, while the rest were given a placebo.
By the halfway point, those taking the drug saw LDL levels fall by roughly 60 percent on average, whereas the placebo group experienced a slight increase.
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After 52 weeks, reductions remained above 55 percent.
Other markers linked to cardiovascular risk, non-HDL cholesterol, apolipoprotein B and lipoprotein(a), also showed meaningful declines.
Although the results parallel what injectable PCSK9 drugs typically achieve, researchers stress that larger studies are needed to determine long-term safety and whether these improvements translate into fewer heart-related events.
Still, experts say the possibility of an effective oral option could broaden access to advanced cholesterol-lowering therapy in the future.
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Sources: Medical News Today, and JAMA.
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This article is made and published by Ida-Marie Palm Varbæk, who may have used AI in the preparation.