Vertex Reports Results of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Two P-III Studies for Cystic Fibrosis
Vertex’s Kalydeco (ivacaftor) Receives Health Canada’s Approval for Cystic Fibrosis (CF) with CFTR Gene Mutation in Children Aged 12 to <24mos.
Shots:
- The two P-III studies involve assessing of Trikafta in 403 & 107 patients aged ≥12yrs. with CF with one F508del mutation and one minimal function mutation (F/MF) in the CFTR gene & two F508del mutations (F/F) for 24wks. & 4wks. respectively
- The two P-II studies resulted in meeting all 1EPs & 2EPs demonstrating significant improvements in lung function and other measures of the disease and is well tolerated. The results from the studies serve as the basis of the US FDA approval and MAA currently under review with EMA
- Trikafta is a prescription therapy used to treat CF in patients aged ≥12yrs. who have at least one copy of the F508del mutation in the CFTR gene with its anticipated global regulatory submission in 2020
Click here to read full press release/ article | Ref: Vertex | Image: Benzinga