CF drug for children in CanadaFebruary 5, 2019
Health Canada has granted market authorization for lumacaftor/ivacaftor (Orkambi) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del CFTR mutation.
Approval is based on a phase 3 open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6-11 years. Improvements in sweat chloride, a secondary endpoint, were observed at week 24.
The phase 3 study also demonstrated changes in key growth parameters, which were also secondary endpoints in the study.
Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is meant to enhance the function of the CFTR protein once it reaches the cell surface.
Orkambi was approved by the US FDA in August of last year for use in patients ages 2 to 5 years who have two copies of the F508del CFTR mutation.
Orkambi is marketed by Vertex Pharmaceuticals Inc.