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
Cramer's Action Alerts PLUS - See his portfolio and get alerts BEFORE every trade. Learn more NOW!

FDA Advisory Committee Votes On Investigational Medicine Metreleptin

AstraZeneca (NYSE:AZN) and Bristol-Myers Squibb Company (NYSE:BMY) today announced the U.S. Food and Drug Administration’s (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) recommends the investigational medicine metreleptin for the treatment of pediatric and adult patients with generalized lipodystrophy (LD). Specifically, EMDAC determined by a vote of 11 to 1 that there is substantial evidence that the benefits of metreleptin exceed the risks for the treatment of pediatric and adult patients with generalized lipodystrophy.

EMDAC did not recommend metreleptin in patients with partial LD for the indication currently proposed, by a vote of 2 to 10. AstraZeneca and Bristol-Myers Squibb remain committed to pursuing metreleptin for treatment in patients with metabolic disorders associated with partial LD. The Companies acknowledge the committee’s feedback and will continue to work with the FDA to identify the appropriate patients with partial LD who may benefit from metreleptin.

The FDA is not bound by the EMDAC’s recommendation but will take it into consideration when reviewing the Biologics License Application (BLA) for metreleptin.

LD is a group of rare syndromes often associated with severe metabolic abnormalities and significant morbidity and mortality. Metreleptin is being reviewed by the FDA as a treatment of pediatric and adult patients with generalized lipodystrophy (LD) or metabolic disorders associated with partial LD, including hypertriglyceridemia and/or diabetes mellitus inadequately controlled on a current therapy, and/or evidence of hepatic steatosis.

The Prescription Drug User Fee Act (PDUFA) goal date for metreleptin is February 24, 2014. There are no therapies approved by the FDA to treat patients with rare forms of LD (not including HIV-associated LD).

The EMDAC based its recommendations on a review of data from the metreleptin clinical development program for LD that supported the BLA submission, including pivotal efficacy and safety data from two open-label, investigator-sponsored National Institutes of Health (NIH) studies, as well as important supplemental efficacy and safety data on investigational metreleptin from an additional open-label expanded access study, FHA101.

1 of 3

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

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
Stocks Under $10

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

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Try it NOW
Only $9.95
Try it NOW
14-Days Free
Try it NOW

Check Out Our Best Services for Investors

Dividend Stock Advisor

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
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Options Profits

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

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
Try it NOW
Try it NOW
Try it NOW
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 18,203.37 -85.26 -0.47%
S&P 500 2,107.78 -9.61 -0.45%
NASDAQ 4,979.9010 -28.1950 -0.56%

Partners Compare Online Brokers

Free Reports

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