Deutivacaftor

The transport of charged ions across cell membranes is normally achieved through the actions of the cystic fibrosis transmembrane regulator (CFTR) protein. This protein acts as a channel and allows for the passage of chloride and sodium. This process affects the movement of water in and out of the tissues and impacts the production of mucus that lubricates and protects certain organs and body tissues, including the lungs. In the F508del mutation of the CFTR gene, one amino acid is deleted at the position 508, therefore, the CFTR channel function is compromised, resulting in thickened mucus secretions.

Deutivacaftor is a CFTR potentiator that works alongside CFTR correctors - like tezacaftor and vanzacaftor - to potentiate open probability (or gating) of the CFTR protein at the cell surface. This facilitates adequate ion channel formation and increased water and salt movement through cell membranes, resulting in a reduction in thickened mucous production.