On 18 May, at the 2026 American Thoracic Society (ATS) international conference, during a poster session, a single-centre, observational cohort study was presented with results for elexacaftor/tezacaftor/ivacaftor (ETI) that focused on clinical and laboratory parameters in people with cystic fibrosis (pwCF) carrying at least one F508del allele.
The therapy, also known by its brand name Trikafta/Kaftrio, is a triple combination therapy from Vertex Pharmaceuticals and is formulated to improve the function of the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. CFTR modulator therapies have been instrumental in transforming the cystic fibrosis (CF) treatment paradigm, particularly demonstrating improvements in lung function and in overall quality of life.
Go deeper with GlobalData
The latest set of positive long-term data presented, with a particular focus on haemoglobin (HbA1c), liver function tests and immunoglobulin G (IgG) levels, will continue to bolster Trikafta’s dominant position in the CF market and strengthen the therapy’s evidence base to enable more informed decision-making and patient monitoring as part of the broader post-authorization efficacy studies and market access framework.
In the single-centre, observational cohort study conducted in Germany, 106 adult pwCF with at least one F508del allele receiving ETI were followed for 48 months. Clinical and biochemical changes were measured, including those related to lung function, pulmonary exacerbations, sweat chloride concentration, and laboratory parameters.
Improvement in lung function was observed from baseline, with a change of 0.5L in forced expiratory volume in one second (FEV1) and 15.6% in percent predicted FEV1. Improvements were also observed in inspiratory vital capacity (IVC) and reduction of pulmonary exacerbations, with an increase of 1.06L and reduction of 28.9%, respectively. Sweat chloride concentration was reduced by 41mmol/L, and HbA1c decreased by 2.31%. Additionally, a reduction of 5.79g/L from baseline in IgG levels was also observed, highlighting easing of chronic inflammation and lowering of bacterial load.
CFTR modulator therapies such as Trikafta have transitioned CF from a life-limiting illness into a manageable, chronic disease. In this regard, mapping the long-term effects of Trikafta on laboratory parameters is of paramount importance to help maintain Trikafta’s position as gold standard among CFTR modulators.
While this therapy primarily targets respiratory symptoms, it also carries a boxed warning highlighting the risks of drug-induced liver injury and liver failure, and therefore the need for continuous monitoring of liver function throughout use. For the same reason, the long-term effects of Trikafta and the wider drug class on hepatic biomarkers are an ongoing area of research. It is this gap that is primarily addressed by long-term, real-world data such as the study presented above.
In addition to reiterating the efficacy of Trikafta in cystic fibrosis, the positive results presented in this research also help establish a formidable benchmark for CFTR modulator therapies, and help inform the clinical framework that is necessary to assess the efficacy of new and upcoming targeted therapies for CF. As the pipeline of next-generation CFTR modulators develops, long-term real-world datasets of this kind will be essential reference points for payers evaluating the comparative benefit and cost-effectiveness of any successor therapy seeking reimbursement in markets where Trikafta has already set a high clinical bar.
