The U.S. Food and Drug Administration has approved a new treatment option for millions of Americans with high cholesterol. This comes in the form of a first-ever once-daily oral PCSK9 inhibitor, a pill that can significantly reduce "bad" cholesterol in the blood.
The medication, known as Lipfendra, blocks the action of PCSK9, a naturally occurring protein in the liver that affects how the body removes LDL cholesterol from the bloodstream. In simple terms, PCSK9 prevents the liver from recycling LDL receptors, leading to increased bad cholesterol in the blood.
High levels of LDL cholesterol are a major risk factor for heart disease, a condition that is often triggered by the buildup of plaques in the arteries. According to experts, this issue is largely a result of the body's evolutionary adaptation to a time when food was scarce, but now poses a significant threat to heart health.
Lipfendra has been shown to be effective in reducing LDL cholesterol by 56% to 60% when combined with statin therapy in phase 3 clinical trials. This is roughly double the impact of statins alone, which are the most commonly prescribed cholesterol-lowering medications.
The pill has also been found to be generally well-tolerated in the trials, with the most common side effects being diarrhea and dizziness. Serious side effects and treatment discontinuations occurred at rates similar to those in the placebo group.
The approval of Lipfendra marks a significant development in the quest to reduce high cholesterol levels in the U.S. population. While statins are effective for many people, some patients may require additional medication to manage their cholesterol levels, especially if they have inherited forms of high cholesterol or experience adverse side effects from statins.
PCSK9 inhibitors, such as Repatha and Praluent, have previously been available only as injections, which may have contributed to their underuse. The oral form of Lipfendra, on the other hand, is well-tolerated and just as effective.
Further research is needed to determine whether Lipfendra also reduces the risk of heart attacks, strokes, and cardiovascular deaths, but results from a large clinical trial are expected to be available in 2029.
