Posted by Michael Wonder on 19 Nov 2018
Vertex receives European CHMP positive opinion for Orkambi (lumacaftor/ivacaftor) for treatment of children with cystic fibrosis aged 2 to 5 years old with most common form of the disease
16 November 2018 - If approved, lumacaftor/ivacaftor will be the first medicine in Europe to treat the underlying cause of cystic fibrosis for approximately 1,500 young children with two copies of the F508del mutation.
Vertex Pharmaceuticals (Europe) Limited today announced that the EMA CHMP adopted a positive opinion for Orkambi (lumacaftor/ivacaftor) for the treatment of people with cystic fibrosis aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease.
The submission was supported by data from 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 in these paediatric patients generally consistent with that in patients aged 6 years and older.
