Panel narrowly backs mipomersen

FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 9-6 that efficacy and safety data for Kynamro mipomersen from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) support approval to treat patients with homozygous familial hypercholesterolemia (hoFH). Panelists were concerned about high discontinuation rates, modest efficacy and hepatic toxicity. Genzyme said it is planning to conduct a postmarketing observational study to monitor long term efficacy and safety of Kynamro, but did not provide details of the study. Of the six panel members who voted against Kynamro, four voted in favor of approving lomitapide from Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) for the same indication during Wednesday's committee meeting (see BioCentury Extra, Oct. 17). ...