This Day On The Street
Continue to site right-arrow
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

FDA Grants Orphan Drug Designation To Aegerion Pharmaceuticals' Drug Candidate, Lomitapide, For Treatment Of Familial Chylomicronemia

Stocks in this article: AEGR

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

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.

1 of 2

Select the service that is right for you!

Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!


DOW 17,804.80 +26.65 0.15%
S&P 500 2,070.65 +9.42 0.46%
NASDAQ 4,765.38 +16.9840 0.36%

Brokerage Partners

Rates from

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs