9-9-13 8:30 AM EDT | Email Article

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 www.aegerion.com.

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 http://www.sec.gov), 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
         amurphy@aegerion.com

Aegerion Pharmaceuticals, Inc. Logo

GlobeNewswire, Inc. 2013
Add a Comment

News   More...

Securities related to this article
Aegerion Pharmaceuticals Inc View aegr Stock Quote
Aegerion Pharmaceuticals, Inc. View aegr Stock Quote
 
Start Premium Trial Register For Free

P F Fund Financial Data (13,000+ funds)
P F Stock Financial Data (7,000+ stocks)
P F Stock and Fund Screeners (basic)
P F Investing Articles and Market Commentary
P F Articles Archive (>30 days)
P F Discuss (dozens of stock, fund, bond, and general bulletin boards)
P F Portfolio Manager (basic)
P F Morningstar Investment Classroom
P F Access Your Portfolio Anytime, Anywhere via Your Mobile Device
P   Morningstar Fund Analyst Reports (full research on 1,700 funds, ETFs, and CEFs)
P   Morningstar Stock Analyst Reports (full research on more than 1,100 stocks)
P   Portfolio Manager (advanced with 10 X-Ray analyses, including recommendations)
P   Portfolio Monitor (monthly and on-demand personalized portfolio statements)
P   Morningstar Proprietary Stock Information (stock star ratings, buy/sell prices, economic moat ratings, and more)
P   Morningstar 5-Star Stock and Fund Favorites & Red Flags eNewsletters
P   Premium Stock and Fund Screeners (advanced with nearly infinite ways to find the best securities for you)
P   Discounts on Morningstar newsletters, books, seminars, and more

Sign-up to Morningstar


Basic Access

Articles and Videos
Morningstar video reports, articles, data, and tools. Dig into our lineup of e-newsletters and investing classroom courses outlining sensible strategies for maximizing your investments.
Morningstar Discuss
Follow the buzz on individual stocks or fund families. Plus, read the latest posts from Morningstar members and analysts.
Portfolio Manager
Track your investments and watch lists with our Portfolio Manager. Customize your view with our comprehensive stock, fund, and ETF data.

Access these features and more when you sign up for Free Membership.
Join Morningstar today. It's Free.

Unlimited Access

Analyst Research
Gather in-depth, actionable insight on over 3,700 stock and mutual funds covered by Morningstar analysts. View our mutual fund Analyst Reports favored by professionals, savvy experienced investors, and new investors researching options for retirement savings, college plans, or other investment needs.
Portfolio X-Ray®
Ensure your portfolio is positioned exactly how you want with Morningstar's X-Ray Interpreter and Stock Intersection tools. A Barron's "Best of the Web" award winner for five years running, X-Ray Interpreter goes under the hood of your portfolio to provide a useful allocation, sector, and geographic analysis. And you can discover how much of a stock you really own, individually and through mutual funds, with Stock Intersection.
Investment Picks
Find new 5-star stocks (greatly undervalued companies) to consider daily. Our 5-star stock rating is a proven winner, consistently beating benchmarks like the S&P 500. Plus, browse our Fund Analyst Picks and Stewardship Grades to find shareholder-friendly mutual funds that are poised for outperformance.
Portfolio Monitor
Get a clear view of your investments with personalized reports about your portfolio. Receive monthly statements and generate reports on-demand to help you stay on track to meet your investment goals.

Access these features and more when you sign up for Premium Membership.
Start your free 14-day trial today online
Your subscription may be tax deductible. Please contact your tax advisor.

Content Partners
Morningstar - 2013/9/9 - Aegerion Pharmaceuticals' Lomitapide Receives Orphan Drug Designation in Japan for the Treatment of Homozygous Familial Hypercholesterolemia
Name |  Ticker |  Star Rating |  Market Cap |  Stock Type |  Sector |  Industry Star Rating |  Investment Style |  Total Assets |  Category |  Top Holdings |  Top Sectors |  Symbol |  Title Star Rating |  Category |  Total Assets |  Top Holdings |  Top Sectors |  Symbol |  Name Topic |  Sector |  Key Indicators |  User Interest |  Market Cap |  Industry Name |  Ticker |  Star Rating |  Market Cap |  Stock Type |  Sector |  Industry Star Rating |  Investment Style |  Total Assets |  Category |  Top Holdings |  Top Sectors |  Symbol / Ticker |  Title Star Rating |  Category |  Total Assets |  Symbol / Ticker |  Name Title |  Date |  Author |  Collection |  Popularity |  Interest Title |  Date |  Company |  Symbol |  Interest |  Popularity Title |  Date |  Company |  Symbol |  Interest |  Popularity Title |  Date |  Author |  Collection |  Interest |  Popularity

Previous: Aegerion Pharmaceuticals' JUXTAPID(TM) (lomitapide) Capsules Approved in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)  |  Next: Aegion Corporation Announces New $10 Million Share Repurchase Plan