This program is studying a combination therapy combining three CFTR modulators. Vanzacaftor (VX-121) and tezacaftor are CFTR correctors, a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Deutivacaftor (VX-561) is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. Deutivacaftor may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.

Status

Two Phase 3 studies evaluating the effectiveness of vanzacaftor/tezacaftor/deutivacaftor compared to Trikafta^® (elexacaftor/tezacaftor/ivacaftor) have been completed. The studies enrolled people with CF ages 12 years and older who have at least one F508del mutation or another mutation responsive to CFTR modulators.

An additional Phase 3, open-label study evaluated the safety and effectiveness of vanzacaftor/tezacaftor/deutivacaftor in children with CF ages 6 to 11 years with at least one mutation responsive to CFTR modulators.

A Phase 3, open-label study is currently underway for children with CF ages 2 to 5 years with at least one mutation responsive to CFTR modulators.

Sponsor

This program is sponsored by Vertex Pharmaceuticals. This program is being conducted within the Therapeutics Development Network (TDN).