Shots:

• The approval is based on two P-III clinical study result assessing Orkambi in 60 patients with CF + 2 copies of F508del mutation- in Children Aged 2 to 5
• The studies demonstrated an improvement in sweat chloride- 2 EP @24 weeks (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7- -27.6- n=49)
• Orkambi is a combination of lumacaftor and ivacaftor and being developed for Cystic Fibrosis. lumacaftor increase the amount of mature protein at the cell surface by targeting the processing and deals with defect of F508del-CFTR protein. Ivacaftor enhance the function of CFTR protein once it reaches the cell surface. It has already received EU approval for the treatment of CF in patients aged ≥ 6yr

Ref: Vertex Pharmaceuticals | Image: Vertex