The U.S. Food and Drug Administration (FDA) has approved Lipfendra (enlicitide), making it the first oral inhibitor of the PCSK9 protein used to lower levels of low-density lipoprotein (LDL) cholesterol in adults with high cholesterol.

Cholesterol lowered by 59% after 24 weeks

The new drug includes treatment for patients with heterozygous familial hypercholesterolemia (HeFH), taken once daily in conjunction with diet and physical activity, targeting the PCSK9 protein which limits the liver's ability to remove LDL cholesterol from the blood, thereby increasing LDL clearance.

The approval represents a significant shift in the treatment of these conditions because all previous drugs in this class were administered only by injection, whereas the new therapy offers a more convenient oral option for patients. It was based on results from a study showing the drug reduced cholesterol by up to 59% after 24 weeks in patients receiving lipid-lowering therapy.

The results indicated the continued efficacy and safety of the drug over a follow-up period of up to one year, with common side effects such as diarrhea, dizziness, and extremity pain. However, its effectiveness in reducing the risk of heart attacks or strokes has not been established. It is recommended to take the tablet on an empty stomach and wait 30 minutes before eating.