Posted by Michael Wonder on 14 Dec 2018
Health Canada grants market authorisation for Orkambi (lumacaftor/ivacaftor) for children with cystic fibrosis aged 2 to 5 years old with most common form of the disease
13 December 2018 - Orkambi is the first medicine in Canada to treat the underlying cause of cystic fibrosis in young children with two copies of the F508del mutation.
Vertex Pharmaceuticals today announced that Health Canada has granted market authorisation for Orkambi (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis 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 Orkambi 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 (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6, n=49).
