Shots:

• 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

Ref: Vertex | Image: Vertex