FDA Approves Sanofi's Kynamro to Treat Rare Cholesterol Disorder

   By Jennifer Corbett Dooren 
 

WASHINGTON--The U.S. Food and Drug Administration Tuesday approved a drug called Kynamro that will be marketed by marketed by Sanofi's (SNY, SNY.FR) Genzyme unit to treat a rare form of a disease that causes very high cholesterol.

The drug is also known by its generic name, mipomersen, and was developed by Isis Pharmaceuticals Inc. (ISIS). The product was approved to treat a condition called homozygous familial hypercholesterolemia, or HoFH, a disorder that causes very high cholesterol, and is meant to be given with other cholesterol-lowering drugs. The FDA estimates HoFH affects one person out of a million people in the U.S. and said people with the disorder often have heart attacks and die before age 30.

Kynamro is an orphan drug approval, meaning it was developed to treat a disorder affecting fewer than 200,000 people in the U.S.

"Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles" that cause high cholesterol, said Eric Colman, an FDA deputy director in the agency's drug evaluation and research center.

The product will carry the FDA's strictest boxed warning discussing risks of liver problems that could cause liver disease.

FDA based its decision primarily on a study that involved 51 patients with HoFH. On average, so-called LDL cholesterol levels, or the "bad" kind of cholesterol, by about 25% during the first six months of treatment.

The FDA said it is also requiring four post-marketing studies for Kynamro including one looking at liver abnormalities in patients being treated with the drug.

-Write to Jennifer Corbett Dooren at [e-mail]

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