CAMBRIDGE, Mass., April 30, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat debilitating and often fatal rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for lomitapide, a once-daily, oral treatment for Homozygous Familial Hypercholesterolemia (HoFH), for review. Aegerion is seeking authorization to market lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies to reduce cholesterol in patients with HoFH. As previously disclosed, the FDA has classified the submission as a standard review with a 10-month Prescription Drug User Fee Act (PDUFA) timetable.
"The FDA's acceptance of our NDA filing for review, on the heels of the EMA's acceptance of our MAA filing in March, is an important step in our plan to bring lomitapide to the market," said Marc D. Beer, Chief Executive Officer. "We look forward to working closely with the FDA and the EMA as each agency continues its review of our respective submissions. We hope to bring lomitapide to the market expeditiously, as we believe that existing treatments leave significant unmet need for patients with this devastating disease."
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.
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.
About Aegerion Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR) is an emerging biopharmaceutical company focused on the development and commercialization of novel, breakthrough therapeutics to treat debilitating and often fatal rare diseases. Aegerion is motivated by its commitment to patients first. We are also attentive to our core values of integrity, innovation, responsibility to healthcare providers, development of employees and always – scientific and clinical excellence.
Forward-Looking Statements
This press release contains forward-looking statements, including statements about the potential for regulatory approval and launch of lomitapide. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, among other factors: the risk that the FDA or other applicable regulatory authorities may ask for additional data, information or studies to be completed or provided prior to approval; the risk that the FDA or other applicable regulatory authorities may not agree with our validation plan or may require additional work related to the commercial manufacturing process to be completed prior to approval or may, in the course of the inspection of manufacturing facilities, identify issues to be resolved; the risk that the FDA or other applicable regulatory authorities may not be satisfied with the safety profile of lomitapide; the risk that we do not receive approval of lomitapide in the US or EU on a timely basis or at all; and the risk that unexpected hurdles, technical issues or data may arise. For additional disclosure regarding these and other risks we face, see the disclosure contained in our public filings with the U.S. Securities and Exchange Commission (available on the SEC's website at http://www.sec.gov), including the "Risk Factors" section of our most recent Annual Report on Form 10-K. The Company undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.