This trial is testing tezacaftor (formerly VX-661) in combination with ivacaftor. Tezacaftor (VX-661) is a compound designed to move the defective CFTR protein to the proper place in the airway cell surface. Ivacaftor helps 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.
A Phase 2 trial for people with CF who have two copies of the F508del mutation was completed January 2015. Several Phase 3 trials are underway in people who have one or two copies of the F508del mutation.
SYMDEKO™ is approved in the USA
This program is sponsored by Vertex Pharmaceuticals Inc. and partially funded by Cystic Fibrosis Foundation Therapeutics. The program is being conducted within CFFT's Therapeutics Development Network.
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