Researchers found that Kalydeco-treated CF patients had a significantly lower risk of death, pulmonary exacerbation, hospitalization, transplant, and prevalence of CF-related complications and pathological microorganisms, as well as improved lung function.
Data was collected from the U.S. and U.K. cystic fibrosis registries and published in the journal Thorax in a study titled, “Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor.”
Kalydeco, commercialized by Vertex Pharmaceuticals, is an oral medicine that targets genetic mutations associated with CF — namely those in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This small molecule is the first of a new class of therapies, called CFTR potentiators, demonstrating clinical benefits in CF patients.
The therapy was first approved in both the U.S. and European Union in 2012 to treat CF patients over the age of 6 who specifically had a G551D mutation in the CFTR gene. Now it is prescribed for the treatment of several other mutations, in a broader spectrum of ages, depending on the mutation and country of residence.
In clinical studies, CF patients treated with Kalydeco had improved lung function, as well as other clinical benefits, such as improved respiratory symptoms and pulmonary exacerbations.
However, little is known about the long-term effects of Kalydeco in CF patients. Since it was developed for chronic, lifelong use, knowing the treatment’s long-term effects is crucial for everyone involved, including patients, caregivers, payers, healthcare providers, and regulators.
In the study, researchers investigated the effects of Kalydeco treatment on disease progression and clinical outcomes of CF patients, using data from the U.S. and U.K. CF registries following the therapy’s commercial availability in the countries.
The study enrolled 1,256 patients who began Kalydeco treatment in 2012 and 6,200 control patients who had never taken Kalydeco from the U.S. registry, and 411 patients who began Kalydeco treatment in 2013 and 2,069 controls from the U.K. registry.
Different outcomes were compared, including risk of death, pulmonary exacerbation, hospitalization, transplant, lung function changes, and prevalence of CF-related complications and pathological microorganisms.
Kalydeco-treated CF patients showed a significantly lower risk of death (0.6% vs. 1.6%), pulmonary exacerbation (27.8% vs. 43.3%), hospitalization (27.5% vs. 43.1%), and transplantation (0.2% vs. 1.1%), compared with controls in the U.S. A similar result was also found in the U.K. registry.
The treated group also had less CF-related complications, such as diabetes and depression, and pathogenic microorganisms. These patients’ lung function also improved after treatment with Kalydeco.
No additional safety concerns for the therapy were reported in this study.
Based on the results, the team concluded, “these observational analyses of the U.S. and U.K. CF registry patient cohorts found no new safety concerns and revealed favorable results in clinically important outcomes, including lower risk of death and organ transplantation among ivacaftor-treated patients, adding to the growing body of literature supporting disease modification by CFTR modulation with ivacaftor.”