Drug Development Pipeline
Elexacaftor + tezacaftor + ivacaftor (Trikafta®)
Restore CFTR Function
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 who are 12 years and older who have at least one copy of the F508del mutation or one of 177 other approved mutations. Click here to see a list of all of the mutations currently approved by the FDA for Trikafta® (see page 8 for a list of approved mutations).
Two phase 3 studies to test the safety and effectiveness of Trikafta™ in people with CF 12 years and older have been completed. People with two copies of the F508del mutation had a 10 percent increase in lung function compared to treatment with the modulator tezacaftor/ivacaftor (Symdeko®), and people with one copy of F508del had more than a 14 percent increase in lung function compared to placebo. In people with one copy of the F508del mutation, Trikafta® was also associated with significant improvements in sweat chloride, pulmonary exacerbations and quality of life.
A study of Trikafta® in children with CF ages 2-5 years old is currently underway.
This program is sponsored by Vertex Pharmaceuticals and partially funded by the Cystic Fibrosis Foundation. This program is being conducted within the Therapeutics Development Network (TDN).
Recent Elexacaftor + tezacaftor + ivacaftor (Trikafta®) Studies
Latest News on Elexacaftor/tezacaftor/ivacaftor
June 09, 2021
The U.S. Food and Drug Administration has approved the use of Trikafta® (elexacaftor/tezacaftor/ivacaftor) for children ages 6 through 11 who have at least one copy of the F508del mutation or certain mutations in the CFTR gene that are responsive based on lab data.
January 26, 2021
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
September 10, 2020
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.
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