A Breakthrough Cholesterol Pill Could Help Millions Struggling With Statins

For decades, there were only two options to bring your LDL cholesterol down. Doctors would prescribe statins and, if those weren't enough, your next option was an injection.

PCSK9 is a protein your liver makes that essentially reduces your body's ability to clear LDL cholesterol from the blood. When the PCSK9 protein is blocked, the liver can pull more LDL out of circulation, and blood levels drop.

The FDA approval comes on the heels of two large, randomized, placebo-controlled trials involving a total of 3,207 adults with severe high cholesterol, all of whom were already taking the maximum tolerated dose of statin therapy.

The first trial enrolled adults who either already had cardiovascular disease or were at high risk for it. Their average LDL at the start was 96 mg/dL. After 24 weeks, those taking Lipfendra saw an average LDL reduction of 56% compared to placebo.

The second trial focused specifically on adults with heterozygous familial hypercholesterolemia (HeFH), an inherited condition that causes high LDL cholesterol. Their average starting LDL was 119 mg/dL. After 24 weeks, the Lipfendra group saw an average reduction of 59% compared to placebo.

Clinical guidance says it's reasonable for adults who are already on the maximum statin dose but still sitting above 70 mg/dL to consider adding a PCSK9 inhibitor2. In the past, these people have had to rely on injections, but a pill that drops LDL 56 to 59% is enough to move many people closer to their cholesterol targets.

This new drug is not a replacement for statins, but an add-on for people who still can't reach their LDL targets with statins alone. It is approved for adults with high cholesterol or HeFH who are already on diet, exercise, and maximally tolerated statin therapy.

While drug developments like this are exciting, medication is one piece of a larger picture.

According to the latest ACC/AHA guidelines on preventing cardiovascular disease3, the most important foundation for preventing cardiovascular disease is a healthy lifestyle, and that comes before pharmacological therapy. The guideline recommends a diet rich in vegetables, fruits, whole grains, and lean protein, along with at least 150 minutes of moderate-intensity physical activity per week.

For many people, those changes are enough to move LDL in the right direction. When they're not enough on their own, statins are typically the next step. And when statins, even at the maximum dose, still leave LDL above target, add-on therapies like Lipfendra come in.

This is the treatment ladder for treating high cholesterol, and Lipfendra sits at the top of it for people who have genuinely worked their way up the other rungs.