Understanding real-world microbiological outcomes will guide long-term CFTR modulator use in CF: GlobalData

At the 2025 European Respiratory Society (ERS) International Congress in Amsterdam, researchers from Dalhousie University and the University of Toronto presented findings from a comprehensive, registry-based analysis examining the long-term clinical and microbiological impacts of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. The study, spanning data from 2019-2023 across Canadian and US cystic fibrosis (CF) registries, revealed sustained clinical improvements alongside complex microbiological trends that have implications for patient monitoring strategies and infection risk stratification, says GlobalData.

Erela Dana, Director, Neurology and Immunology at GlobalData, comments, “While the study demonstrated expected clinical benefits with sustained improvements in lung function metrics, the microbiological landscape proved more complex than anticipated. Most notably, the analysis revealed a 300 per cent relative increase in nontuberculous mycobacteria (NTM) detections among non-hospitalised adults receiving CFTR therapy between 2019 and 2023.”

The retrospective cohort analysis focused on patients with more than 12 months of continuous modulator use, evaluating lung function changes and trends in key respiratory pathogens. The temporal analysis highlighted variable responses across patient subgroups, with adults showing more favorable microbiological outcomes compared to pediatrics, and inconsistent pathogen clearance patterns despite improved CFTR function.

Dana adds, “As modulator therapy becomes lifelong treatment for an increasing number of CF patients, understanding the evolution of airway microbiology becomes critical for preventing long-term complications. The findings underscore the complexity of CFTR restoration’s impact on the CF airway ecosystem and suggest that current microbiological monitoring frameworks may need adaptation.”

According to GlobalData’s Cystic Fibrosis market forecast report, CFTR modulators accounted for 87.5 per cent of CF treatment sales in 2024 across the seven major markets (7MM*) and are projected to maintain dominant market share through 2034. The CF market is projected to reach $16.0 billion across the 7MM by 2034.

Dana concludes, “Future CF therapies will need to demonstrate not only functional improvements but also favorable long-term microbiological profiles to succeed in this treatment landscape. As registry-based surveillance becomes more prevalent in clinical development programs, understanding real-world microbiological outcomes will guide evidence-based prescribing decisions in an increasingly outcomes-driven CF landscape.”

*7MM- The US, France, Germany, Italy, Spain, the UK, and Canada