New Rx Treats Genetic Form of Severely High Cholesterol

The US Food and Drug Administration (FDA) has approved a new medication to treat severely high cholesterol caused by a genetic condition.

The FDA approved Evkeeza (evinacumab-dgnb) as an add-on therapy to treat homozygous familial hypercholesterolemia (HoFH). Approved for use in patients who are 12 years old or older, Evkeeza comes in the form of an injection to be given under the skin.

HoFH is a genetic condition that causes severely high cholesterol. According to the FDA, this condition affects around 1 in 250,000 people. In those it affects, HoFH causes extremely high levels of low-density lipoprotein cholesterol (LDL-C), known as “bad cholesterol.”

These high levels of cholesterol can cause heart attacks and heart disease in patients when they are in their teens or 20s. Many patients who do not get effective treatment die before they turn 30, the FDA noted.

Evkeeza is meant to prevent the high levels of cholesterol that make this disease so deadly. According to the FDA, this drug blocks a protein that slows down the processing of certain fats in the body. By doing this, Evkeeza speeds up the body's ability to break down the fats that cause high cholesterol.

The FDA assessed the effectiveness and safety of Evkeeza through a 24-week trial of 65 patients with HoFH. Patients who received Evkeeza saw their LDL-C levels decrease by 47 percent on average. Those who received a placebo saw an average increase of 2 percent.

The FDA noted that Evkeeza's ability to reduce cholesterol levels caused by other issues or prevent death has not been studied.

Side effects of Evkeeza included cold, flu-like illness, nausea and runny nose. There were some serious allergic reactions to this drug in clinical trials, the FDA noted. And Evkeeza may cause harm to unborn babies.

Talk to your health care provider about which treatments are available for your condition.

The FDA approved Evkeeza for Regeneron Pharmaceuticals, Inc.