Will Pfizer Challenge Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) Cystic Fibrosis Empire ?

November 21, 2012

       Pfizer announces on November 19, 2012, that the company is to receive up to $58 million in a 6-year pre-clinical R&D program, from Cystic Fibrosis FoundationTherapeutics (CFFT).  The program is to develop new drugs for the most common mutation of the disease – the Delta F508 mutation. CFFT is the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation (CFF). 

       Almost 90% of CF patients have at least 1 copy of the Delta F508 mutation. This mutation prevents a protein from maintaining the proper flow of salt and fluids into airways, causing thick secretions to form.

      The Cystic Fibrosis (CF) Foundation gave $75 million to Vertex Pharmaceuticals for the “collaboration of scientific, clinical, and financial support for the development of orphan drug Kalydeco (Ivacaftor)”. 

      Vertex Pharmaceuticals reports on November 1, 2012, at their 3^rd Quarter 2012 financial results meeting, 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 

Cystic Fibrosis Foundation Drug Development Pipeline  

PharmaPhorum.com November 20, 2012 article titled, “Cystic Fibrosis Foundation to invest up to $58m in Pfizer partnership” 

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