Vertex Pharmaceuticals Incorporated (Vertex) announced that 10 abstracts from its cystic fibrosis (CF) R&D program will be presented at the 26th Annual North American Cystic Fibrosis Conference (NACFC) in Orlando, Florida, from October 11–13, 2012. Previously announced data from a Phase II study of VX-809 combined with ivacaftor in people with the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del, will be presented for the first time. Additional data from Vertex’s work to discover and develop medicines that target the underlying cause of CF will also be presented, including data on Kalydeco (ivacaftor) in people with CF who have the G551D mutation.
Titles of the abstracts:
• An abstract entitled, 'Hyperpolarized Gas MRI of Ivacaftor Therapy in Persons with Cystic Fibrosis and the G551D-CFTR Mutation.' will be presented on October 11, 2012.
• An abstract entitled, '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,' will be presented on October 11, 2012
• Abstract: 'Identification and Characterization of CFTR Corrector VRT-534 (C-18).'
• Abstract: 'Ivacaftor Potentiates Multiple Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Forms.'
• Abstract: 'Long-Term Safety and Efficacy of Ivacaftor in Persons with Cystic Fibrosis who have the G551D-CFTR Mutation.'
• Abstract: 'Patient-Reported Outcomes in Phase 3 Trials of Ivacaftor in Subjects with CF who have the G551D-CFTR Mutation.'
• Abstract: 'Nutritional Status Measures Among Persons with CF Carrying the G551D-CFTR Mutation who Received Ivacaftor or Placebo in Phase 3 Clinical Trials.'
• Abstract: '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.'
• Abstract: 'Exposure-Response Relationship for FEV1 and Sweat Chloride in Patients with Cystic Fibrosis Treated with Ivacaftor, a CFTR Potentiator.'
• Abstract: 'Clinical Pharmacology Profile of Ivacaftor, a CFTR Potentiator.'
Kalydeco is the first treatment to target the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, it is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. Kalydeco (150mg, q12h) was first approved by the FDA in January 2012 and by the European Medicines Agency in July 2012, for use in people with CF ages six and older who have at least one copy of the G551D mutation in the CFTR gene.
Vertex retains worldwide rights to develop and commercialize Kalydeco. It is under priority review by the Therapeutic Product Directorate (TPD) of Health Canada, and an application for review has been submitted to the Therapeutic Goods Administration (TGA) of Australia.