Drug Development Pipeline
Tezacaftor + ivacaftor (Symdeko®)
Restore CFTR Function
Tezacaftor + ivacaftor (Symdeko®) is a combination therapy combining tezacaftor, a compound designed to move the defective CFTR protein to the proper place in the airway cell surface, with ivacaftor, which 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.
Tezacaftor/ivacaftor (Symdeko®) is approved for individuals 12 years and older with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 153 specified mutations -- regardless of their other mutation. Click here to see a list of all of the mutations currently approved by the FDA for Symdeko® (see page 8 for a list of approved mutations).
A phase 2 study for people with CF who have two copies of the F508del mutation was completed January 2015. Two phase 3 studies of tezacaftor in combination with ivacaftor showed positive results in March 2017. Additional studies are currently underway.
This program is sponsored by Vertex Pharmaceuticals Inc. and partially funded by the Cystic Fibrosis Foundation. The program is being conducted within the Therapeutics Development Network.
Recent Tezacaftor + ivacaftor (Symdeko®) Studies
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