March 8, 2012

Aegerion Pharmaceuticals Comments on Standard Review Classification for Lomitapide NDA

CAMBRIDGE, Mass., March 8, 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 commented on U.S. Food and Drug Administration (FDA)'s standard review classification of Aegerion's New Drug Application (NDA) for its lead investigational therapeutic, lomitapide.

Lomitapide is a small molecule, microsomal triglyceride transfer protein inhibitor, or MTP-I, in development as a once-daily oral therapeutic for use as an adjunct to a low-fat diet and other lipid-lowering therapies to reduce cholesterol in patients with Homozygous Familial Hypercholesterolemia (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.

"After discussions with the FDA, we are comfortable with the justification for the standard review classification," said Marc D. Beer, Aegerion's Chief Executive Officer. "The FDA provided a clear and executable pathway for Aegerion to file an NDA based upon 56-week data from a single, open label, 78-week Phase III clinical trial in 29 patients with HoFH, with a primary endpoint of LDL-C reduction. In light of this surrogate endpoint, the NDA has been given a standard review classification as it is not feasible to study cardiovascular outcomes in the HoFH patient population. We appreciate the FDA's willingness to inform us of this classification decision early, and look forward to taking the additional time provided under standard review to thoroughly prepare for advisory committee and the planned commercial launch of lomitapide to patients in need."

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 not agree with our validation plan or may require additional work related to the commercial manufacturing process to be completed prior to approval or launch; the risk that the FDA or other applicable regulatory authorities 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 US 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 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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