March 5, 2012

Aegerion Pharmaceuticals Submits Marketing Applications for Lomitapide in U.S. and EU

CAMBRIDGE, Mass., March 5, 2012 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, today announced that it has submitted a New Drug Application (NDA) to the U.S. 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 U.S. and EU. The company plans to initiate a clinical study of lomitapide for the treatment of FC in 2012.

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 the potential for regulatory approval and launch of lomitapide, and plans for further clinical trials. 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 the Company's regulatory filings may not be accepted by the applicable regulatory authorities or that such acceptance may be delayed; the risk that 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 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 FDA or other applicable regulatory authorities may not be satisfied with the safety profile of lomitapide; the risk that the Company does not receive approval of lomitapide in the U.S. or EU on a timely basis or at all; and the risk that unexpected hurdles, technical issues or data may delay further clinical trials in FC. 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 Quarterly Report on Form 10-Q 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.

CONTACT: Aegerion Pharmaceuticals, Inc.

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