FDA panel backs Aegerion's lomitapide
FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 13-2 that efficacy and safety data for lomitapide from Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) support approval to treat homozygous familial hypercholesterolemia (hoFH). Aegerion agreed to conduct a long-term postmarketing observational study of lomitapide that would monitor drug effects on liver enzymes and hepatotoxicity in 300 patients, which represents almost half of the estimated 750 hoFH patients in the U.S. and EU. FDA recommended eight years of follow-up for the trial. Panelists suggested the study also collect data on anatomical changes related to atherosclerosis, as well as track CV events and cancer prevalence. Trading in Aegerion's stock was halted on Wednesday. The small molecule microsomal triglyceride transfer protein (MTP) inhibitor has a Dec. 29 PDUFA date. ...