FH Treatments

Statins have been the go-to-medication to treat individuals with familial hypercholesterolemia (FH) and other forms of high cholesterol since the 1980s. But a new therapy, called PCSK9 Inhibitors, are now recognized as an effective way to manage FH.

Approved by the Food and Drug Administration (FDA) in 2015, PCSK9 Inhibitors are injectable drugs that target proprotein convertase subtilisin kexin 9 (also known as PCSK9), a protein that interferes with the clearance of blood cholesterol.

The drugs work by disabling PCSK9, which allows LDL-cholesterol (bad cholesterol) to be removed from the blood by the normal liver clearance processes.

PCSK9 inhibitors for Familial Hypercholesterolemia

What types of PCSK9 inhibitors are Available?

There are currently two PCSK9 Inhibitors available today in the United States:

  • Alirocumab (Praluent)
  • Evolocumab (Repatha)

PCSK9 inhibitors, in combination with other LDL lowering drugs, have been demonstrated to be highly effective for some people. Studies have shown that these drugs reduce LDL- cholesterol by 40 to 65 percent beyond the effect of other lipid lowering drugs.  Use of a PCSK9 inhibitor can reduce the chance of heart attack and stroke.

For individuals with FH, this is an important breakthrough, as early and regular treatment can reduce the risk of stroke and heart attacks significantly. New PCSK9 inhibitors are in development and currently in clinical trials.

PCSK9 Inhibitor Trial Results

PCSK9 Inhibitor Trial Results

The Odyssey study showed PCSK9 inhibitors improved outcomes.

Read more

When Are PCSK9 inhibitors Recommended for Familial Hypercholesterolemia?

Doctors will prescribe statins, cholesterol absorption inhibitors or bile acid sequestrants first. If these therapies alone, or in combination with each other, don’t lower cholesterol enough, PCSK9 inhibitors may be used.


  • PCSK9 inhibitors are approved for use, in addition to diet and maximally tolerated statin therapy, in adult patients with heterozygous familial hypercholesterolemia (HeFH), or clinical atherosclerotic cardiovascular disease (ASCVD), such as heart attacks or strokes, who require additional lowering of LDL cholesterol.



A lipid specialist can provide more information on the use and effectiveness of PCSK9 Inhibitors, and whether the new class of drugs may be right for you.

Need Help Navigating Insurance

Do you have questions on how to get approval for a prescription? Are you experiencing insurance denials? Our Navigating Insurance guide will guide you through the entire process.
Navigating Insurance Guide - FH Foundation