FDA approves Genzyme drug for rare cholesterol disorder

Kynamro approved for HoFH

SILVER SPRING, Md. — The Food and Drug Administration has approved a new drug for treating a rare cholesterol disorder, the agency said.

The FDA announced the approval of Kynamro (mipomersen sodium), made by Genzyme for the treatment of homozygous familial hypercholesterolemia, or HoFH. Kynamro has been approved as an orphan drug, a designation the FDA gives to drugs that treat diseases affecting fewer than 200,000 people.

The disease is an inherited condition affecting about one-in-1 million people in the United States and prevents the body from removing low-density lipoprotein cholesterol from the bloodstream. For people with HoFH, heart attacks and death often occur before age 30.

"Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles that ultimately give rise to LDL-C," FDA Division of Metabolism and Endocrinology Products deputy director Eric Colman said.

The FDA approved another drug for HoFH, Aegerion Pharmaceuticals' Juxtapid (lomitapide), in December 2012.

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