Aegerion Pharmaceuticals, Inc., an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA), and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval of Aegerion's lead investigational therapeutic, lomitapide, as an adjunct to a low fat diet and other lipid-lowering therapies to reduce cholesterol in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Lomitapide is a small molecule, microsomal triglyceride transfer protein inhibitor, or MTP-I, in development as a once-daily oral therapeutic for the treatment of HoFH. HoFH is a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C) cholesterol in the blood. Patients diagnosed with HoFH typically have as much as three to six times the normal amount of LDL-C while on a variety of lipid-lowering drug treatments, putting them at risk for a major cardiovascular event.
"The submission of our NDA and MAA filings represents a significant corporate accomplishment," said Marc D. Beer, chief executive officer. "In 2011, we assembled a team of experts with deep experience in orphan and genetic diseases, dedicated to working with the worldwide HoFH community. This is an important step towards making lomitapide available to patients suffering from HoFH who currently have inadequate treatment options."
Lomitapide holds orphan drug designation for the treatment of HoFH in the United States, and for the treatment of familial chylomicronemia (FC) in the US and EU. The company plans to initiate a clinical study of lomitapide for the treatment of FC in 2012.