Drug OK'd for deadly genetic condition tied to cholesterol

(HealthDay)—Kynamro (mipomersen sodium) has been approved by the U.S. Food and Drug Administration to treat a rare inherited condition in which the body can't remove low-density lipoprotein (LDL) cholesterol from the blood.

LDL is the so-called "bad" cholesterol that can clog the arteries and cause heart attack and stroke. Many people with homozygous familial (HoFH) have a heart attack and die before age 30, the FDA said in a news release.

HoFH affects approximately one of every 1 million people in the United States. Kynamro is a once-weekly injection designed to lower creation of particles that ultimately form LDL, the agency said.

The drug was clinically evaluated among 51 people with HoFH. Among Kynamro users, LDL levels fell an average of about 25 percent during the first 26 weeks, the FDA said. The drug will carry a "black box" label warning of possible liver abnormalities that could lead to progressive .

More common side effects noted during clinical testing included injection-site reactions, flu-like symptoms, nausea, headache and elevated liver enzymes.

Kynamro is produced by Genzyme Corp., of Cambridge, Mass.

More information: To learn more about high cholesterol, visit the U.S. National Library of Medicine.

add to favorites email to friend print save as pdf

Related Stories

FDA approves juxtapid for rare cholesterol disorder

Dec 27, 2012

(HealthDay)—The U.S. Food and Drug Administration has approved the orphan drug Juxtapid (lomitapide) for patients with homozygous familial hypercholesterolemia (HoFH), for use in combination with a low-fat ...

Canadian scientists discover cause of high cholesterol

Oct 28, 2012

Canadian scientists have discovered that a protein called resistin, secreted by fat tissue, causes high levels of "bad" cholesterol (low-density lipoprotein or LDL), increasing the risk of heart disease.

Recommended for you

Big cities take aim at prescription painkillers

Sep 16, 2014

Some of the nation's largest cities are ratcheting up their criticism of prescription painkillers, blaming the industry for a wave of addiction and overdoses that have ravaged their communities and busted local budgets.

World Health Organization policy improves use of medicines

Sep 16, 2014

In this issue of PLOS Medicine, Kathleen Holloway from WHO and David Henry (University of Toronto, Canada) evaluated data on reported adherence to WHO essential medicines practices and measures of quality use of medicines from 5 ...

User comments