Phase III data on Kalydeco (Vertex) shows benefits for Cystic Fibrosis patients

A Phase III study of Kalydeco (ivacaftor), from Vertex, for patients with Cystic Fibrosis shows the drug significantly improves lung function and reduces pulmonary exacerbations. The STRIVE study randomised 161 patients aged 12 and over with at least one copy of the G551D mutation in the CFTR gene to ivacaftor (150mg every 12 hours) or placebo. Results showed a mean absolute improvement of 10.6% in predicted FEV1 after 24 weeks' treatment with ivacaftor compared to placebo (p<0.0001), sustained at 10.5% at 48 weeks.>

The ENVISION study, which included 52 children aged 6-11 years, showed similar absolute improvement of 12.5% in predicted FEV1 with ivacaftor at 24 weeks compared to placebo (p<0.0001), with a 10.0% improvement maintained at 48 weeks. strive showed a significant reduction in pulmonary exacerbations and clinically significant improvement in the respiratory domain of patients' quality of life with ivacaftor. both studies showed an increase in body weight (mean increases of 2.7kg at 48 weeks in strive and 2.8kg in envision).>

Results presented at the European Respiratory Society's Annual Congress by Dr Edward McKone, Consultant Respiratory Physician at St Vincent's University Hospital, Dublin, Ireland, from the follow-up PERSIST study showed that improvements in lung function with ivacaftor were sustained for patients continuing treatment from the STRIVE and ENVISION trials for up to 96 weeks.