Aegerion Pharmaceuticals Inc., an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, has commented on US Food and Drug Administration (FDA)'s standard review classification of Aegerion's New Drug Application (NDA) for its lead investigational therapeutic, lomitapide.
Lomitapide is a small molecule, microsomal triglyceride transfer protein inhibitor, or MTP-I, in development as a once-daily oral therapeutic for use as an adjunct to a low-fat diet and other lipid-lowering therapies to reduce cholesterol in patients with Homozygous Familial Hypercholesterolemia (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.
“After discussions with the FDA, we are comfortable with the justification for the standard review classification,” said Marc D Beer, Aegerion's CEO. “The FDA provided a clear and executable pathway for Aegerion to file an NDA based upon 56-week data from a single, open label, 78-week phase III clinical trial in 29 patients with HoFH, with a primary endpoint of LDL-C reduction. In light of this surrogate endpoint, the NDA has been given a standard review classification as it is not feasible to study cardiovascular outcomes in the HoFH patient population. We appreciate the FDA's willingness to inform us of this classification decision early, and look forward to taking the additional time provided under standard review to thoroughly prepare for advisory committee and the planned commercial launch of lomitapide to patients in need.”
Lomitapide holds orphan drug designation for the treatment of HoFH in the United States, and for the treatment of familial chylomicronemia (FC) in the U.S. and EU. The company plans to initiate a clinical study of lomitapide for the treatment of FC in 2012.
Aegerion Pharmaceuticals, Inc. aspires to change the way rare, genetic lipid disorders are treated and focused on the development and commercialization of novel life-altering therapeutics for debilitating and often fatal orphan diseases.