This Day On The Street
Continue to site right-arrow
ADVERTISEMENT
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!

Vertex Submits Supplemental New Drug Application (sNDA) To U.S. Food And Drug Administration For KALYDECO™ (ivacaftor) Monotherapy For People With Non-G551D Gating Mutations

Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of KALYDECO TM (ivacaftor) monotherapy for people with cystic fibrosis (CF) ages 6 and older who have at least one non-G551D gating mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene. KALYDECO is currently approved for people with CF ages 6 and older who have at least one copy of the G551D mutation. CF is caused by a defective CFTR protein that results from mutations in the CFTR gene. G551D is known as a gating mutation, and there are an additional nine known gating mutations. Gating mutations prevent the CFTR protein from opening or working (gating) properly at the cell surface. Worldwide, approximately 2,000 people with CF ages 6 and older have at least one copy of the G551D mutation, and approximately 400 people with CF ages 6 and older have at least one non-G551D gating mutation.

"Today’s submission to the FDA is an important step toward our goal of helping more people with CF," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “The study in people with non-G551D gating mutations is the first of multiple ongoing efforts to expand the number of people who may benefit from ivacaftor, and we expect additional data from these ongoing studies beginning later this year.”

The sNDA submission is based on previously announced data from a Phase 3 study of ivacaftor monotherapy that showed statistically significant improvements in lung function (FEV 1). The mean absolute treatment difference in percent predicted FEV 1 between treatment with ivacaftor and placebo was 10.7% (p < 0.0001) and the mean relative treatment difference in percent predicted FEV 1 was 14.2% (p < 0.0001) through the 8-week treatment period. The safety and tolerability results observed in this study were consistent with those observed in prior Phase 3 studies of ivacaftor monotherapy in people with CF who have the G551D mutation. The study in gating mutations is one of three ongoing Phase 3 label-expansion studies designed to evaluate whether additional people with CF may benefit from treatment with ivacaftor alone.

In addition to the sNDA submission, Vertex intends to submit a Marketing Authorization Application (MAA) variation in Europe in October 2013 for people with CF ages 6 and older who have at least one non-G551D gating mutation.

1 of 5

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!
SYM TRADE IT LAST %CHG

Markets

DOW 18,135.72 +38.82 0.21%
S&P 500 2,101.04 +2.51 0.12%
NASDAQ 4,982.8090 +15.6680 0.32%

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