FDA Expands Repatha Approval for Adults at Risk of Heart Problems Due to High Cholesterol and Approves Use as Standalone Therapy in Adults and Children with Homozygous Familial Hypercholesterolemia

Medically Reviewed by Amy Hemstreet, PharmD on August 26, 2025
2 min read

Repatha (evolocumab) is an injectable medicine that helps lower levels of low-density lipoprotein cholesterol (LDL), often called “bad” cholesterol. Approved in 2015, Repatha can now be used in adults with poorly controlled LDL who haven’t been diagnosed with heart disease but are at high risk for serious heart problems such as stroke, heart attack, and death. Until now, Repatha was approved for lowering LDL in adults with diagnosed heart disease.

The recent approval also allows Repatha to be used as a standalone treatment in adults and children ages 10 and older with a rare genetic form of high cholesterol known as homozygous familial hypercholesterolemia (HoFH). It is also approved for use in adults and children ages 10 and older for another genetic cholesterol disorder, called heterozygous familial hypercholesterolemia (HeFH).

Repatha belongs to a group of medicines known as PCSK9 inhibitors. It helps your liver remove more LDL cholesterol from your blood and lower your overall cholesterol levels. Repatha should be used along with diet and exercise.

High LDL levels are one of the main risk factors for heart disease, which is the leading cause of death in the U.S. This expanded approval allows Repatha to be used earlier to help prevent these events in people who are at risk and whose cholesterol levels haven’t responded well to diet, exercise, or statins alone. 

In more than 50 studies spanning over 15 years, Repatha has shown to be effective in lowering LDL levels and reducing the risk for heart-related events in people with high LDL, inherited cholesterol-related conditions, or who couldn’t take statins. In these studies, people taking Repatha had fewer heart attacks, strokes, and other major heart-related events compared to those who took a placebo, even when they were already taking statins.

The most common side effects are runny nose, sore throat, symptoms of the common cold, flu or flu-like symptoms, back pain, high blood sugar levels (diabetes), and redness, pain, or bruising from the injection. Some people had serious allergic reactions.

 

Repatha is given as an injection under the skin. You may take this medicine every two weeks or once a month, depending on what your health care provider recommends. Each injection device is for one-time use only and should be thrown away in a sharps container.

You should not use Repatha if you’ve had a serious allergic reaction to evolocumab or any of its ingredients. Signs of a serious reaction include rash; trouble breathing; or swelling of the face, lips, tongue, throat, or arms. If you notice any of these, stop using the medicine and contact your health care provider or call 911 right away.

Tell your health care provider if you are allergic to rubber or latex, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

This content was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.