Vertex has presented long-term, observational data highlighting the benefit of Kalydeco in patients with cystic fibrosis.

Real-world data from two large, independent cystic fibrosis (CF) registries was used to compare patients treated with the drug - a cystic fibrosis transmembrane (CFTR) modulator - to a comparator group of matched patients who had never received such treatment.

According to the firm, observational studies show that patients treated with Kalydeco (ivacaftor) had fewer deaths, transplantations, hospitalisations and pulmonary exacerbations, compared to patients matched on age, sex and disease severity as assessed by genotype who had never received a CFTR modulator.

The results, it said, were consistent with all previous annual analyses performed since the first year of commercial availability of the medicine, which was four years in the UK and five years in the US.

In the analysis of the 2016 data, a significantly smaller proportion of treated patients in the US died (0.4 percent vs 1.0 percent) or had transplantations (0.3 percent vs 1.1 percent) versus the comparator arm.

Similar trends were observed in the UK although this was not statistically significant (death 0.4 percent vs 0.9 percent; transplantation 0.2 percent vs 0.9 percent), the firm noted.

In both registries, treated patients also had significantly lower risks of pulmonary exacerbations (PEx) and hospitalisations, compared to comparator.

In the UK data, hospitalisation for PEx was 24.5 percent vs 43.3 percent, and PEx were 31.6 percent vs 54.5 percent. In the US data, hospitalisation for any cause was 23.9 percent vs 35.6 percent and PEx 23.3 percent vs 32.4 percent.

“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,” noted Professor Diana Bilton, study author and Adjunct Professor, NHLI Imperial College, London & Hon Consultant, Royal Brompton Hospital, London.

“These studies support the idea that CFTR modulator therapy is able to modify the course of the disease in cystic fibrosis patients.”