FDA Advisory Committee recommends lomitapide (Aegerion Pharma) for HoFH

In a 13-2 vote, the FDA Endocrinologic and Metabolic Drugs Advisory considered that Aegerion Pharma provided sufficient efficacy and safety data to support marketing of lomitapide for Homozygous Familial Hypercholesterolemia (HoFH), despite its association with liver toxicity. The application was based on a single 18-month open-label, single-arm pivotal Phase III trial, which only enrolled 29 patients with HoFH, with supportive data from an extension study and a proof-of-concept Phase II study involving six patients.