Aegerion Pharmaceuticals Submits US & EU Marketing Applications
Aegerion Pharmaceuticals, Inc. recently announced it has submitted an NDA to the US FDA and an MAA to the 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, a rare genetic lipid disorder resulting in an accumulation of (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 toward 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
Aegerion Pharmaceuticals, Inc. aspires to change the way rare, genetic lipid disorders are treated. It is an emerging biopharmaceutical company focused on the development and commercialization of novel life-altering therapeutics for debilitating and often fatal orphan diseases. For more information, visit http://www.aegerion.com.
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