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Why Is The Upcoming Week Important For Vertex Pharmaceuticals’ Orphan Drug Kalydeco For Cystic Fibrosis ?

October 8, 2012

       An October 5, 2012 article from discusses Vertex Pharmaceuticals important presentation at the 2012 North American Cystic Fibrosis Conference (NACF) for Thursday, October 11, 2012 in Orlando, Florida.  The presentation will be on the final data from the Phase II study of VX-809 in combination with orphan drug Kalydeco for Cystic Fibrosis (CF) patients with the F508del mutation – 40% of CF patients. Per this article VX-809 + Kalydeco,” … could generate $3 – 4 billion in sales (on top of the $1 billion or so in peak sales of Kalydeco monotherapy in the much smaller G551D mutation patient population). 

        Adam Feuerstein, the author of the article, is going to the NACF conference (Orphan Druganaut September 26, 2012 Blog Post) to cover this presentation and also Vertex Pharmaceutical’s investor meeting. A decision has already been made to move the VX-809 + Kalydeco combination into a Phase III study to start in early 2013.

          The Phase II study of VX-809 + Kalydeco had a lot of controversy over the summer with investors who were angered when Vertex Pharmaceuticals lowered the interim results. This resulted in a shareholder class action complaint against Vertex Pharmaceuticals (Orphan Druganaut September 8, 2012 Blog Post).         

Copyright © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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