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Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) And Cystic Fibrosis Development Programs

November 2, 2012

       On November 1, 2012, Vertex Pharmaceuticals reports on 3rd Q 2012 financial results and the status of their Cystic Fibrosis (CF) Development Programs. 

        Vertex Pharmaceuticals reports the following for their CF Development Programs : 

1)  Three Phase 3 label expansion trials

     –  Phase 3 study of Kalydeco currently ongoing in patients with CF, 6 years and older, and have at least 1 copy of the R117H mutation – an estimated 3% of US CF patients have at least 1 R117H mutation 

     –  Phase 3 study of Kalydeco currently ongoing in patients with CF, 6 years and older, and who have at least 1 non-G551D CFTR gating mutation – an estimated 1% of US CF patients have at least 1 non-G551D gating mutation 

      –  Phase 3 study of Kalydeco recently initiated in children with CF, 2 – 5 years of age, and who have a gating mutation; enrollment to start by end of 2012. 

2)  One Phase 2 study underway for Kalydeco monotherapy

     –  Phase 2 proof-of-concept study evaluating Kalydeco in people with clinical evidence of residual CFTR function – an estimated 5 – 10% of US CF people may have residual CFTR function.       

3)  Study to start at beginning of 2013 to evaluate combination therapy of VX-809 and Kalydeco, in patients with CF, ages 12 and older, and with 2 copies of the F508del mutation – most common form of CF. 

4)  To identify additional CF treatment regimens in collaboration with the CF Foundation Therapeutics. 

References 

ClinicalTrials.gov for “Kalydeco AND Vertex Pharmaceuticals”

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

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