- The approval is based on P-III EVOLVE & EXPAND studies result assessing Symdeko vs PBO in 750 patients with CF having two copies of the F508del mutation or with one F508del mutation & residual CFTR activity aged> 12yrs.
- The P-III EVOLVE & EXPAND studies demonstrated improvement in lung function, mean absolute change in ppFEV1 (4.0% vs 6.8%) and was well tolerated. It was published in New England Journal of Medicine in Nov’17
- Symdeko is a combination therapy of tezacaftor/ivacaftor + ivacaftor and has received US FDA’s, Health Canada, EU’s approval in Feb’18, Jun’18 & Oct’18 respectively for the same indication
Click here to read full press release/ article | Ref: Vertex | Image: Business Wire