Shots:

• The approval is based on the results of two P-III studies EVOLVE & EXPAND assessing Symkevi + Kalydeco vs PBO in 750 patients with CF having two copies of the F508del mutation or with one F508del mutation & a copy of either of 14mutations showing CFTR residual activity
• P-III EVOLVE & EXPAND results: mean absolute change in ppFEV1 (4.0%- 6.8%); well tolerated. The combination is approved by the US FDA & Health Canada in Feb- 2018 & Mar- 2018 respectively and is marketed as Symdeko in the US and Canada
• Symkevi is developed to detect mutations in CFTR gene. Additionally- EMA has recommended to maintain the Orphan Drug Designation for the combination

Ref: Vertex Pharmaceutical| Image: Vertex