FDA advisory panel votes 9 to 6 to approve Genzyme's Kynamro

The US Food and Drug Administration’s Endocrinologic and Metabolic Drugs Advisory yesterday voted by nine to six that Kynamro (mipomersen sodium) should be approved for the treatment of patients with homozygous familial hypercholesterolemia (HoFH), though expressing some safety concerns. The drug is being developed by Sanofi (Euronext: SAN) subsidiary Genzyme in partnership with Isis Pharmaceuticals (Nasdaq: ISIS).

The majority recommendation was reached despite misgivings about side effects, including liver problems and a potentially higher risk for cancer. HoFH patients treated with Kynamro saw their low-density lipoprotein cholesterol levels drop nearly 22% further than those given a placebo. However, some panel members called that a "moderate" improvement and said it was unclear how effective the drug might be at reducing bad cholesterol to medically acceptable levels or helping HoFH patients avoid cardiovascular disease. Robert Smith of Brown University, who voted against recommendation, said the drug's ability to help some patients was not enough to outweigh potential.

Development of neoplasms noted