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 26 specified mutations -- regardless of their other mutation.
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
Latest News on VX-661/ivacaftor
November 27, 2018
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
October 31, 2017
The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of lumacaftor/ivacaftor (Orkambi®) and ivacaftor/tezacaftor (VX-661). The Foundation is sharing input throughout the process to help incorporate the patient and clinical perspective.
July 18, 2017
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
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