The U.S. Food and Drug Administration (FDA) has approved Lipfendra (enlicitide), the first once-daily oral medication in the PCSK9 inhibitor class, designed to lower LDL cholesterol. Merck, the pharmaceutical company behind the drug, announced the approval on Thursday. Lipfendra is intended for adults with high cholesterol, including inherited forms, and is to be used in conjunction with diet and other cholesterol-lowering therapies.
How Lipfendra Works
Lipfendra targets the PCSK9 protein, a naturally occurring substance that influences how the liver removes LDL, or "bad," cholesterol from the blood. By blocking PCSK9, the medication facilitates the increased removal of LDL cholesterol by the liver. Dr. Marc Siegel, a Fox News senior medical analyst, explained that this protein typically prevents the recycling of LDL receptors in the liver, leading to elevated levels of bad cholesterol. In modern diets, this can contribute to plaque buildup and heart disease.
Clinical Trial Results
Phase 3 clinical trials demonstrated that Lipfendra, when used as an add-on to statin therapy, reduced LDL cholesterol levels by approximately 56% to 60%. Siegel noted that this reduction is nearly double the impact typically seen with statins alone. The drug was generally well-tolerated by participants. The most common side effects reported were diarrhea and dizziness. Serious side effects and instances where participants discontinued treatment occurred at rates comparable to the placebo group.
Statins are the most frequently prescribed medications for cholesterol management. They function by inhibiting a liver enzyme involved in cholesterol production. While effective for many, some individuals with very high cholesterol, genetic predispositions to high cholesterol, or those who experience adverse effects from statins may require additional treatments, such as PCSK9 inhibitors.
A New Option for Patients
Previously, PCSK9 inhibitors were only available in injectable forms, such as Repatha and Praluent. Experts suggest that the injection method may have contributed to their underutilization. Siegel stated that the oral form, Lipfendra, is as effective and well-tolerated as its injectable counterparts.
Further research is ongoing to determine Lipfendra's impact on reducing the risk of heart attacks, strokes, and cardiovascular deaths. Merck anticipates results from a large clinical trial in 2029. Steve Nissen, head of preventive cardiology at the Cleveland Clinic, has indicated that lowering LDL cholesterol as much as possible is crucial, particularly for individuals at risk for heart disease.