Drug Development Pipeline
Elexacaftor + tezacaftor + ivacaftor (Trikafta®)
Restore CFTR Protein
Elexacaftor + tezacaftor + ivacaftor (Trikafta®) is a combination therapy combining three CFTR modulators. Elexacaftor 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. Ivacaftor is a potentiator. Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
Trikafta is approved for people with CF ages 2 and older who have at least one copy of the F508del mutation or at least one copy of 177 specified mutations (Esta lista de mutaciones también está disponible en español).
An ongoing observational study will look at the effects of Trikafta on growth in young children with CF.
This program is sponsored by Vertex Pharmaceuticals and partially funded by the Cystic Fibrosis Foundation. This program was conducted within the Therapeutics Development Network (TDN).
Recent Elexacaftor + tezacaftor + ivacaftor (Trikafta®) Studies
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