CAMBRIDGE, Mass., Sept. 28, 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 today that European regulators confirmed filing requirements for the Company's Marketing Authorization Application (MAA) for its drug candidate, lomitapide, for the treatment of homozygous familial hypercholesterolemia (HoFH). The Company met with the European Medicines Agency (EMA) as well as with its Rapporteur and Co-Rapporteur and their respective agencies.
The European regulators confirmed that Aegerion's ongoing 29 patient single Phase III clinical study design is considered acceptable and that the anticipated non-clinical and clinical data package will be adequate for the MAA filing. The Company now expects to file its MAA submission in the first quarter of 2012, to comply with the regulators' recommended data requirements to assess the stability of lomitapide bulk drug substance.
"All three assessment bodies confirmed the adequacy of the nonclinical and clinical data package as the basis for the MAA filing for HoFH, which was very important validation that the Company's regulatory strategy in the EU is sound," commented Marc D. Beer, Chief Executive Officer of Aegerion Pharmaceuticals. "Based on the input received, we plan to gather stability data on three batches of lomitapide drug substance of a longer duration than is currently required for our planned NDA filing, necessitating a three month shift in our target MAA filing date. Our priority is to take all necessary steps to maximize the potential for approval of this life-saving drug in the European community."
Separately, the Company reiterated its focus on producing the highest quality NDA filing possible. As such, the Company is revising its previously targeted NDA filing date from the end of this year to the first quarter of 2012. "Our goal is to make lomitapide available to all patients of need worldwide," said Mr. Beer. "We will expeditiously file in each region the highest quality applications in an effort to achieve this goal."
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 in Phase III clinical development. Lomitapide is initially being developed to treat patients with a rare genetic lipid disorder called homozygous familial hypercholesterolemia, or HoFH. 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 the ongoing development of the Company's product candidates and the expected timing of regulatory filings. 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: our history of operating losses; our potential need for additional capital to fund operations and develop our product candidates; uncertainties associated with the clinical development and associated regulatory filings of our product candidates, including the risk that our regulatory filings may not be accepted by the applicable regulatory authorities or that such acceptance may be delayed, the risk that our 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 our product candidates may be narrower than we expect; risks associated with undesirable side effects experienced by some patients in clinical trials for our product candidates; risks associated with our lack of sales and marketing experience; the highly competitive industry in which we operate; risks associated with our intellectual property rights and the extent to which such intellectual property rights protect our product candidates; the risk that third parties may allege that we infringe their intellectual property rights or that we have failed to comply with the provisions of our in-license agreements; risks associated with our reliance on third parties, in particular clinical research organizations and contract manufacturers; risks associated with our ability to recruit, hire and retain qualified personnel; risks associated with our reliance on certain key personnel; and risks associated with volatility in our 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 the SEC's website at http://www.sec.gov.