If FDA follows its advisory panel's recommendation, Aegerion Pharmaceuticals Inc. and Sanofi's Genzyme Corp. unit could each be launching new drugs to treat the ultra-Orphan disease homozygous familial hypercholesterolemia early next year. But neither the panel discussion nor the companies' clinical trials give doctors much of an idea of how and when to use one drug over the other.

The 15 members of the Endocrinologic and Metabolic Drugs Advisory Committee included only one doctor who treats hoFH patients. The remaining members included hepatologists brought in to discuss elevations in hepatic enzymes and hepatic fat seen with both compounds, as well as statisticians, epidemiologists, endocrinologists, cardiologists and a gastroenterologist.