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Notable Approval: FDA Approves First Oral Therapy that Inhibits Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) to Lower Bad Cholesterol in Adults with High Cholesterol
FDA has approved Lipfendra (enlicitide) as a treatment to be used with diet and exercise, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with high cholesterol or who have an inherited type of high cholesterol called heterozygous familial hypercholesterolemia (HeFH).
Lipfendra is a tablet taken by mouth one time per day.
Condition
Hypercholesterolemia occurs when you have too much low-density lipoprotein cholesterol (LDL-C) or "bad" cholesterol in your blood. Cholesterol is a waxy, fat-like substance that your body needs to build cells and make hormones, but too much LDL-C can be harmful. Over time, excess LDL-C penetrates the lining of your arteries. Together with inflammatory cells, it becomes trapped inside the artery walls, forming plaques. This buildup narrows the arteries and restricts blood flow. If a plaque ruptures, it can trigger a blood clot, potentially leading to serious health problems such as heart attack or stroke.
Early on, high cholesterol usually does not cause symptoms, so many people don't know they have it unless they have a blood test called a lipid panel. The type of food someone eats, low levels of physical activity, excess body weight, and genetics can contribute to high cholesterol levels.
There are many options to treat high cholesterol including statins, ezetimibe, and PCSK9 inhibitors. However, Lipfendra is the first drug that is taken by mouth that blocks PCSK9.
Data Supporting Lipfendra
The efficacy and safety of Lipfendra were demonstrated in two randomized, double-blind, placebo-controlled trials (NCT05952856 and NCT05952869) in a total of 3,207 adults with severe hypercholesterolemia, including those with and without HeFH, who were already taking maximally tolerated statin therapy. The primary endpoint for both trials was the percent change from baseline to Week 24 in LDL-C, compared to placebo.
In the first trial, which included adults with a history of atherosclerotic cardiovascular disease (ASCVD) or were at high risk for ASCVD, the average baseline LDL-C was 96 mg/dL. The average percentage change in LDL-C from baseline to Week 24 in the Lipfendra group was -56% compared to the placebo group.
In the second trial, which only enrolled adults with HeFH, the average baseline LDL-C was 119 mg/dL. The average percentage change in LDL-C from baseline to Week 24 in the Lipfendra group was -59% compared to the placebo group.
Safety Information
In the first trial, the frequency of adverse reactions was similar between those treated with Lipfendra and those receiving placebo. In the second trial, the most common adverse reactions in adults with HeFH treated with Lipfendra that occurred at higher frequencies than placebo were diarrhea and dizziness. In both trials, similar proportions of Lipfendra treated patients and placebo-treated patients discontinued treatment because of adverse reactions.
Designation
Lipfendra received Priority Review for this indication. The approval was granted to Merck Sharp & Dohme LLC.
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