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
jennifer.corbett@dowjones.com
Subscribe to WSJ: http://online.wsj.com?mod=djnwires