The FDA said today that it had granted approval to the novel cholesterol-lowering drug mipomersen sodium for use as an adjunct to diet and drug therapy in patients with homozygous hypercholesterolemia. The drug, which was developed by Isis Pharmaceuticals, will be marketed under the brand name of Kynamro by Genzyme.
Kynamro was approved as an orphan drug, which the FDA describes as meaning it was developed to treat a condition affecting less than 200,000 people. The standard of approval for orphan drugs is less rigorous than other drugs. There has been some confusion about the number of people who have homozygous familial hypercholesterolemia. The FDA and ISIS/Genzyme said the condition affects about one in every million people.