Vanzacaftor-tezacaftor-deutivacaftor, a novel therapeutic, once-daily drug, demonstrates noninferiority to elexacaftor-tezacaftor-ivacaftor (the twice-daily, standard-of-care drug) in lung function improvement with an acceptable safety profile in patients with cystic fibrosis.

"Vanzacaftor-tezacaftor-deutivacaftor is noninferior to elexacaftor-tezacaftor-ivacaftor in terms of FEV% predicted and is safe and well tolerated. Once-daily dosing with vanzacaftor-tezacaftor-deutivacaftor reduces treatment burden, potentially improving adherence, compared with the twice-daily regimen of the current standard of care," the authors wrote.

The study was led by Claire Keating, MD, Columbia University Irving Medical Center, New York City. It was published online on January 01, 2025, in The Lancet Respiratory Medicine.

The efficacy and safety of the new drug combination were compared with those of elexacaftor-tezacaftor-ivacaftor, and not a placebo, after a 4-week treatment period, thus limiting the assessment in patients either unable to tolerate or naive to elexacaftor-tezacaftor-ivacaftor. The study population also lacked ethnic and racial diversity, potentially limiting the generalizability of the findings. Some experiments related to lung function could not be performed due to technical challenges in detecting modest changes in older patients with more severe airway obstruction.

This study was funded by Vertex Pharmaceuticals. Some authors reported being employees of Vertex Pharmaceuticals and owning stock or stock options in that company. Some other authors reported receiving grants, consulting fees, speaker fees, and travel support from various organizations, including Vertex Pharmaceuticals.