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
TheStreet Open House

Raptor Pharmaceutical Corp. Provides Update On PROCYSBI(TM) NDA Review

NOVATO, Calif., Dec. 21, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced that the U.S. Food and Drug Administration ("FDA") will require additional time to complete its review of the New Drug Application ("NDA") for RP103 (PROCYSBI™), for the potential treatment of nephropathic cystinosis. In a notice received today from the FDA, the initial Prescription Drug User Fee Act ("PDUFA") goal date has been extended from January 30, 2013 to April 30, 2013. The three month extension is a standard extension period.

The FDA exercised its option to extend the PDUFA goal date to provide more time to complete the review of information submitted within three months of the PDUFA date. The agency has not asked for additional studies.

About Cystinosis and RP103

Cystinosis is a rare, life-threatening metabolic disorder that causes systemic toxic cystine accumulation. Toxic cystine accumulation leads to progressive and irreversible tissue damage and multi-organ failure including renal failure, blindness, CNS damage, respiratory deficiencies, muscle wasting, and premature death. Cystinosis is usually diagnosed in the first years of life and requires lifelong therapy. Left untreated, the disease is fatal by the end of the first decade of life. Daily cystine depletion is the primary treatment strategy for cystinosis. Despite a life-long requirement for cystine-depleting agent use to help preserve multi-organ function, a cumulative inability to maintain adequate cystine control results in the rapid deterioration of kidney function and ensuing need for kidney transplantation.

RP103 is an oral, delayed and extended-release medication containing enteric-coated spheronized micro-beads of cysteamine bitartrate. PROCYSBI™ is the branded name of RP103 for the potential treatment of cystinosis. The NDA and Marketing Authorization Application for RP103 for the potential treatment of nephropathic cystinosis have been submitted to the FDA and the European Medicines Agency, respectively, and the NDA has been assigned a PDUFA date of April 30, 2013. RP103 is also in clinical development for the potential treatment of Huntington's disease and non-alcoholic steatohepatitis ("NASH"). In cystinosis patients, RP103 may reduce cellular toxicity by continuously removing cystine from the lysosome. RP103, which can also cross the blood-brain barrier, was engineered specifically to allow release of cysteamine bitartrate micro-spheres in the duodenum for optimal absorption while simultaneously enabling administration every 12-hours for the potential treatment of cystinosis.

1 of 2

Select the service that is right for you!

COMPARE ALL SERVICES
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 17,279.74 +13.75 0.08%
S&P 500 2,010.40 -0.96 -0.05%
NASDAQ 4,579.7890 -13.6380 -0.30%

Brokerage Partners

Rates from Bankrate.com

  • 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