Aegerion Pharmaceuticals' Lomitapide Receives Orphan Drug Designation In Japan For The Treatment Of Homozygous Familial Hypercholesterolemia

CAMBRIDGE, Mass., Sept. 9, 2013 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), a biopharmaceutical company dedicated to the development and commercialization of novel, life-altering therapies for patients with debilitating, often fatal, rare diseases, announced today that the company has received orphan drug designation from Japan's Ministry of Labour, Health and Welfare for lomitapide for the treatment of homozygous familial hypercholesterolemia (HoFH).

"Japan's recognition of lomitapide as an orphan drug is important as we continue the ongoing clinical development work required for a future Japanese new drug application," said Marc Beer, Aegerion's Chief Executive Officer. "Japan is a significant market for Aegerion, and we look forward to the potential to treat Japanese HoFH patients in need of therapy."

Aegerion is currently conducting a pharmacokinetic/pharmacodynamic (PK/PD) study of lomitapide required by the Japanese regulatory authorities, expected to be completed in the fourth quarter of 2013. The Company then plans to conduct a small therapeutic study of lomitapide in Japanese adult HoFH patients.

In Japan, drugs and medical devices can be designated as orphan drugs or medical devices based on Article 77-2 of the Pharmaceutical Affairs Law if they are intended for use in fewer than 50,000 patients in Japan and there is a high medical need. Specific measures to support the development of orphan drugs include prioritized consultation regarding clinical development and priority review of applications, reduced application fees, extended registration validity period, financial assistance to help cover research and development expenditures, and tax incentives.

About Aegerion Pharmaceuticals

Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative, life-altering therapies for patients with debilitating, often fatal, rare diseases. For more information about the company, please visit

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the potential for filing of a new drug application in Japan; the possibility of a future approval and commercialization of lomitapide in Japan; the importance of the Japanese market; and statements regarding the Company's future clinical plans. 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 our 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 we may not be successful in completing the PK/PD study and a clinical trial that generates results sufficient to support filing a new drug application for lomitapide in the treatment of adult HoFH in Japan; the risk that, even if we file a new drug application for lomitapide in the treatment of adult HoFH in Japan, regulatory authorities in Japan may not agree that the benefit/risk profile supports approval; the risk that, even if lomitapide is approved in Japan, we may not be able to obtain pricing approval and reimbursement at acceptable levels, or governmental authorities may impose other requirements or restrictions that limit the commercial potential of lomitapide in Japan; and the risk that lomitapide may not gain market acceptance in Japan. For additional disclosure regarding these and other risks we face, see the disclosure contained in our public filings with the U.S. Securities and Exchange Commission (available on the SEC's website at, including the "Risk Factors" section of our most recent Quarterly Report on Form 10-Q. We undertake 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: Investor Contact:         Aegerion Pharmaceuticals, Inc.         Amanda Murphy, Manager, IR         (857) 242-5024

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