FDA Advisory Panel Recommends Aegerion’s Lomitapide to Treat HoFH

Despite liver toxicity concerns, the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 13–2 to recommend approval of Aegerion Pharmaceuticals’ lomitapide. The drug is under review to treat adult patients with homozygous familial hypercholesterolemia (HoFH), a genetic disorder. Panelists expressed numerous concerns about the potential for liver toxicities, and whether the drug might be used off-label for patients with heterozygous familial hypercholesterolemia or for pediatric HoFH patients.  
Drug Industry Daily