Positive long-term data for Trikafta, that was presented at the ATS 2026 International Conference, reinforces the therapy’s efficacy in adult cystic fibrosis (CF) patients with at least one F508del allele. The findings highlighted improvements in clinical and biochemical parameters, strengthening Trikafta’s leadership in the CF market and supporting informed patient monitoring and broader post-authorization market access efforts, according to GlobalData, a leading intelligence and productivity platform.  

In the study conducted in Germany, 106 adult pwCF with at least one F508del allele receiving Trikafta, also known by its generic name,Elexacaftor/Tezacaftor/Ivacaftor (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.

Meaningful improvements in lung function were observed from baseline across both volumetric and percentage-predicted measures of forced expiratory volume in one second (FEV1), alongside improvements in inspiratory vital capacity and reduction in pulmonary exacerbation frequency. Sweat chloride concentration reduced from baseline, consistent with effective modulation of the underlying protein defect.

HbA1c levels also decreased meaningfully, reflecting improvements in metabolic control that are increasingly recognised as an important dimension of long-term CF management. Additionally, IgG levels also declined from baseline indicating an easing of chronic systemic inflammation and bacterial load.

Vinie Varkey, Associate Director for Immunology at GlobalData, comments: “Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies such as Trikafta have been instrumental in transforming the CF treatment paradigm, particularly demonstrating improvements in lung function and in overall quality of life. 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.”

However, while this therapy primarily targets respiratory symptoms, it carries a boxed warning highlighting the risks of drug-induced liver injury and liver failure, and therefore there is a need for continuous monitoring of liver function throughout use.

Varkey continues: “The long-term effects of Trikafta and the wider drug class on hepatic biomarkers are an ongoing area of research, and it is this gap that is primarily addressed by long-term, real-world data such as this German study.”

Looking ahead, in addition to reiterating the efficacy of Trikafta in cystic fibrosis, the positive results presented in this research can also help establish a benchmark for novel CFTR modulator therapies, and help inform the clinical framework that is necessary to assess the efficacy of new and upcoming targeted therapies for CF.

Varkey concludes: “As the pipeline of next-generation CFTR modulators develops, long-term real-world datasets of this kind will be essential reference points for regulators and payers evaluating the comparative benefit and cost-effectiveness of any successor therapy seeking approval and reimbursement in markets where Trikafta has already set a high clinical bar.”