Real-world data from Vertex showing benefit of Kalydeco for cystic fibrosis

Patients treated with Kalydeco (ivacaftor), a cystic fibrosis transmembrane (CFTR) modulator, were compared with a group of patients matched on age, sex and disease severity as assessed by genotype who had never received a CFTR modulator, using real-world data from two independent CF registries. Fewer hospitalisations, pulmonary exacerbations, transplantations and deaths were observed in those patients treated with Kalydeco.
UK data showed 0.4% deaths in treated patients compared with 0.9% in untreated patients, and a ratio of 0.2% to 0.9% respectively for transplantation. Hospitalisation for pulmonary exacerbations was 24.5% compared to 43.3%, and pulmonary exacerbations were 31.6% compared to 54.5% in untreated patients.
Professor Diana Bilton, study author and Adjunct Professor, NHLI Imperial College, London & Hon Consultant, Royal Brompton Hospital, London said: “Although these are observational studies, which have limitations in terms of how they should be interpreted, we saw a range of important benefits for patients over time with ivacaftor treatment, compared to the comparator. These studies support the idea that CFTR modulator therapy is able to modify the course of the disease in cystic fibrosis patients.”