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Vertex Pharmaceuticals, Orphan Drug Kalydeco (Ivacaftor), And New Trial Data To Be Presented At NACFC

September 26, 2012

       Per a September 25, 2012 Vertex Pharmaceuticals Press Release, 10 abstracts from the company’s Cystic Fibrosis (CF) research and development program are to be presented at the 26th Annual North American Cystic Fibrosis Conference (NACFC) in Orlando, Florida, October 11 – 13, 2012.  

        Vertex Pharmaceuticals is to present the following that is of special interest :

–      Previously announced data from Phase 2 study of VX-809 + orphan drug Kalydeco in patients with the most common mutation in the CFTR gene, F508del

–     Data  to be presented on Kalydeco in patients with CF who have the G551D mutation.

           The 10 accepted abstracts are now available on the NACFC website :

1. “Hyperpolarized Gas MRI of Ivacaftor Therapy in Persons with Cystic Fibrosis and the G551D-CFTR Mutation.” Poster #196

2. “The Investigational CFTR Corrector, VX-809 (Lumacaftor) Co-Administered with the Oral Potentiator Ivacaftor Improved CFTR and Lung Function in F508DEL Homozygous Patients: Phase II Study Results.” Poster #260

3. “Identification and Characterization of CFTR Corrector VRT-534 (C-18).” Poster #30.

4. “Ivacaftor Potentiates Multiple Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Forms.” Poster #31.  

5. “Long-Term Safety and Efficacy of Ivacaftor in Persons with Cystic Fibrosis who have the G551D-CFTR Mutation.” Poster #211.  

6. “Patient-Reported Outcomes in Phase 3 Trials of Ivacaftor in Subjects with CF who have the G551D-CFTR Mutation.” Poster #212.

7. “Nutritional Status Measures Among Persons with CF Carrying the G551D-CFTR Mutation who Received Ivacaftor or Placebo in Phase 3 Clinical Trials.” Poster #214.

8. “Lung Clearance Index to Evaluate the Effect of Ivacaftor on Lung Function in Subjects with CF who have the G551D-CFTR Mutation and Mild Lung Disease.” Poster #249.  

9. “Exposure-Response Relationship for FEV1 and Sweat Chloride in Patients with Cystic Fibrosis Treated with Ivacaftor, a CFTR Potentiator.” Poster #235.  

10. “Clinical Pharmacology Profile of Ivacaftor, a CFTR Potentiator.” Poster #236.

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

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