CAMBRIDGE, Mass., March 20, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat rare diseases, today announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for lomitapide, a once-daily, oral treatment for Homozygous Familial Hypercholesterolemia (HoFH). Acceptance of the MAA starts the EMA's review process. 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.
"We look forward to working with the European regulators as they begin their review of the lomitapide MAA," said Marc D. Beer, Chief Executive Officer. "This is an important milestone as we seek to bring lomitapide to the market to address the unmet need that exists for these patients."
Lomitapide will be evaluated through the EU's Centralized Procedure with an official start date of March 21, 2012. If approved, lomitapide will gain marketing authorization for all EU Member States simultaneously.
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) aspires to change the way rare, genetic lipid disorders are treated. We are an emerging biopharmaceutical company focused on the development and commercialization of novel life-altering therapeutics for debilitating and often fatal orphan 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 which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements regarding 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 the Company's 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 applicable regulatory authorities may ask for additional data, information or studies to be completed or provided prior to approval; the risk that 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 risks that the applicable regulatory authorities may not be satisfied with the safety profile of lomitapide; and the risk that the Company does not receive approval of lomitapide on a timely basis or at all. For additional disclosure regarding these and other risks faced by the Company, see the disclosure contained in the Company's public filings with the Securities and Exchange Commission, including the Company's most recent Annual Report on Form 10-K under the heading "Risk Factors" and available on the SEC's website at http://www.sec.gov. 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.