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FDA Grants Orphan Drug Designation to Aegerion Pharmaceuticals' Drug Candidate, Lomitapide, for Treatment of Familial Chylomicronemia

/ Source: GlobeNewswire

Lomitapide, in Phase III Development, Holds Orphan Drug Status for Treatment of

CAMBRIDGE, Mass., March 15, 2011 (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, announced it has received orphan drug designation from the U.S. Food and Drug Administration's (FDA) Office of Orphan Product Development for its lead drug candidate, lomitapide, for the treatment of familial chylomicronemia (FC). Lomitapide is currently being evaluated in an ongoing pivotal Phase III clinical trial for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), and the Company expects to launch a separate clinical trial to evaluate lomitapide as a treatment for FC in the second half of 2011.

"The FDA's grant of orphan drug designation to lomitapide for the treatment of FC represents an important milestone in the overall development and commercialization plan for the compound, and it underscores the unmet need that exists. With this announcement, we are another step closer towards our goal of treating patients with these rare lipid disorders that currently have inadequate treatment options available," commented Marc D. Beer, Chief Executive Officer of Aegerion.

Familial chylomicronemia, a rare genetic disorder and severe form of hypertriglyceridemia that results in extremely high levels of triglycerides in the blood, is the second disease for which Aegerion is developing lomitapide and for which it plans to seek regulatory approval if development is successful. Lomitapide has already been granted orphan drug designation for the treatment of FC in the European Union, and for the treatment of HoFH, a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C) in the blood, in the United States.

Lomitapide is a small molecule microsomal triglyceride transfer protein inhibitor being developed as an oral, once-a-day treatment for patients with severe lipid disorders. Lomitapide is being evaluated for its ability to reduce LDL-C levels in patients with HoFH and reduce triglyceride levels in patients with FC. It reduces lipid levels in the blood by preventing the liver and intestines from secreting lipids into the blood stream.

In the United States, under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition, which is generally a disease that affects fewer than 200,000 individuals in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA requirements. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not change or shorten the duration of the regulatory review and approval process.

About Aegerion Pharmaceuticals, Inc.

Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR) is an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders. The Company's lead product, lomitapide, is initially being developed to treat patients with a rare genetic lipid disorder called homozygous familial hypercholesterolemia, or HoFH, and is currently in Phase III development for this indication The Company also plans to initiate a clinical program for lomitapide to treat patients with a severe genetic form of hypertriglyceridemia called familial chylomicronemia.

Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Private Securities Litigation Reform Act of 1995, including statements regarding expected regulatory filings for and commercialization of the Company's lead product candidate, lomitapide. The forward-looking statements in this release do not constitute guarantees of future performance. These statements are neither promises nor guarantees, 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 things: the Company's history of operating losses; the Company's potential need for additional capital to fund operations and develop its product candidates; uncertainties associated with the clinical development and associated regulatory filings of the Company's product candidates, including the risk that the Company's regulatory filings may not be accepted by the applicable regulatory authorities, the risk that the Company's product candidates may not be approved for any indication, or if approved, the risk that the finally approved definition of the targeted patient populations for the Company's product candidates may be narrower than expected; risks associated with undesirable side effects experienced by some patients in clinical trials for the Company's product candidates; risks associated with the Company's lack of sales and marketing experience; the highly competitive industry in which the Company operates; risks associated with the Company's intellectual property rights and the extent to which such intellectual property rights protect the Company's product candidates; the risk that third parties may allege that the Company infringes their intellectual property rights or that the Company has failed to comply with the provisions of its in-license agreements; risks associated with the Company's reliance on third parties, in particular clinical research organizations and contract manufacturers; risks associated with the Company's ability to recruit, hire and retain qualified personnel; and risks associated with volatility in the Company's stock price as a newly public company. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. 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. 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 its investor relations website at and on the SEC's website at .

CONTACT: Aegerion Pharmaceuticals, Inc. Corporate Will Lewis, President +1 (908) 704-1300 LaVoie Group, Inc. Investors & Media Amanda Murphy +1 (978) 745-4200 x107