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 ages 12 years and older who have at least one copy of the F508del mutation.
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 6-11 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
October 21, 2019
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
October 15, 2019
The Institute for Clinical and Economic Review (ICER) recently posted a draft scoping document to outline their planned assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The document is open for public comment through October 21.
September 13, 2019
The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The first opportunity to comment runs through Sept. 25.
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Explore clinical trials
Be a part of the movement transforming the future of cystic fibrosis treatment.Learn More