Development Programs in Orphan Diseases

Aegerion has several different ongoing and planned adult and pediatric clinical programs designed to evaluate our orphan drug candidate in rare genetic lipid disorders. Currently we are conducting clinical trials to determine the safety and efficacy of our lead candidate lomitapide. Lomitapide is a small molecule, MTP-I inhibitor being developed as an oral, once-a-day treatment for rare orphan diseases. Aegerion has completed a pivotal, Phase III clinical trial to determine the efficacy and long-term safety of lomitapide for the treatment of adult patients with homozygous familial hypercholesterolemia, or HoFH. The Company also plans to initiate clinical trials to treat pediatric patients with HoFH and adult patients with a severe genetic form of hypertriglyceridemia called familial chylomicronemia (FC).