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. CTP-656 may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.
A phase 2 study was conducted to test the safety and effectiveness of VX-561 in adults with CF who have a CFTR gating mutation.
VX-561 is also being tested in combination with two correctors, VX-121 and tezacaftor.
This program is sponsored by Vertex Pharmaceuticals. It is being conducted within the Therapeutics Development Network.
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