Recent data on outcomes following treatment with cystic fibrosis transmembrane conductance regulator (CFTR) modulators add to the growing body of evidence that a reduced level of sweat chloride (SwCL) is associated with improved clinical outcomes, particularly in young people with cystic fibrosis (CF).
Vertex Pharmaceuticals Incorporated presented multiple abstracts highlighting the benefits of Alyftrek at the North American Cystic Fibrosis Conference in Seattle. Several of the abstracts’ demonstrated that improvement in CFTR function, as measured by reduction in SwCL, helps improve outcomes in CF patients. Alyftrek has shown greater reductions in SwCL compared to Trikafta in phase 3 trials. Both therapies led to similar stabilization of lung function and had comparable safety profiles.
The new research suggests Alyftrek has the potential for even greater improvements in quality of life and other health-related outcomes.
Previous phase 3 and open-label extension studies that included patients treated with Alyftrek showed that those who began CFTR modulator therapy at young ages achieved greater CFTR function. Among children who began treatment between six and 11 years of age, those who achieved lower SwCL levels experience broader clinical benefits, including greater gains in lung function, easing of respiratory symptoms, improved nutritional status and lower annual rates of pulmonary exacerbations.
Alyftrek is indicated for the treatment of CF in patients six years of age or older who have an F508del mutation or another responsive mutation in the CFTR gene. The next-generation triple combination (vanzacaftor/tezacaftor/deutivacaftor) drug was approved by the U.S. Food and Drug Administration (FDA) in December 2024.
Currently, the median age of death for people with CF is in their 30s, but projected survival rates are improving as new therapies emerge, such as CFTR modulator therapy.
