New Pill Cuts LDL Cholesterol by 55% in Genetic Disorder Trial

A new drug called enlicitide has shown promising results in lowering LDL cholesterol. Published in JAMA on 9 November, the study focused on patients with heterozygous familial hyperlipidemia—a genetic condition that causes high cholesterol levels. Unlike existing treatments, this medication comes in pill form rather than an injection.

The trial tracked participants over 52 weeks. Those taking enlicitide daily saw their LDL cholesterol drop by an average of 55.3%. In contrast, the placebo group experienced an 8.7% increase. After just 24 weeks, 58.2% of patients on the drug achieved an even sharper reduction of 58.2%.

The drug also improved other key lipid markers. Non-HDL cholesterol, apolipoprotein B, and lipoprotein(a) levels all decreased in the enlicitide group. These findings suggest broader benefits beyond LDL cholesterol alone.

So far, testing has only involved people with heterozygous familial hyperlipidemia. Researchers now plan to study its effects on individuals without the condition. Larger and longer trials will be needed to confirm its safety and effectiveness across a wider population.

Enlicitide marks a potential shift in cholesterol treatment, offering a pill-based alternative to injectable PCSK9 inhibitors. While early results are encouraging, further research will determine its long-term impact. The drug's ability to lower multiple lipid markers could make it a valuable option for patients struggling with high cholesterol.