Shots:

• The approval is based on P-III study results assessing Orkambi in 60 patients with CF- who have two copies of the F508del CFTR mutation aged 6 to 11 years for 24 wks.
• The P-III study demonstrated in reduction of sweat chloride by 31.7 mmol/L and was well-tolerated- Presented at European Cystic Fibrosis Society Conference in June 2018
• Orkambi is a combination of lumacaftor + ivacaftor used for targeting F508del CFTR protein and is approved by the US FDA for CF aged 2 to 5 yrs. in Aug- 2018

Ref: Vertex Pharmaceuticals | Image: Vertex