How Does TRIKAFTA™ Work?

See How TRIKAFTA Works

Watch how the 3 components of TRIKAFTA work together to target the underlying cause. 

See How TRIKAFTA Works

Watch how the 3 components of TRIKAFTA work together to target the underlying cause. 

The Underlying Cause

Cystic fibrosis (CF) is caused by mutations in the CF gene. These mutations lead to defects in a specific protein called the cystic fibrosis transmembrane conductance regulator (CFTR) protein. As a result of these defects, the CFTR proteins don’t work the way they should. The most common mutation is the F508del mutation. 

The F508del mutation causes both defects illustrated below:

Defect 1: Fewer CFTR proteins get to the cell surface, where they are normally located
Defect 2: CFTR proteins don't open up if they do reach the cell surface

Because of these defects, chloride ions cannot move into or out of the cells like they should. This can cause thick, sticky mucus to build up in organs, such as the lungs.

TRIKAFTA: Three Components That Work Together to Target the Underlying Cause

What is known about how TRIKAFTA works was learned from studies conducted in a laboratory. Keep in mind that results from laboratory studies do not always match how these medicines work in a person. If you have questions about your treatment, speak with your healthcare provider.

TRIKAFTA adds elexacaftor to tezacaftor and ivacaftor to target CFTR protein defects caused by the F508del mutation.

 

Mechanism of action of TRIKAFTA

Together, the 3 components help F508del-CFTR proteins function better.

Leah, age 14
F508del/F508del
Leah, age 14
F508del/F508del