Health Canada expands Orkambi label for the treatment of cystic fibrosis for children ages between one and two
Health Canada has granted Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) a marketing authorisation for the treatment of cystic fibrosis (CF) for children ages between one and two
The treatment will now be available to children with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Orkambi became Canada's first disease-modifying CF medicine available to patients between the ages of one and two.
As per Vertex, there are approximately 30 children eligible for this treatment in Canada.
In Trial 7, a multicentre 24-week, open-label study safety in 46 patients between the ages of one and two, Orkambi’s safety profile was found to be similar to that in a similar Phase III trial (NCT03125395) for patients of ages two and older. The results showed that one patient experienced a respiratory-related adverse event (AE) but resumed treatment, one discontinued treatment due to a liver-related AE, and five had serious AEs which were considered to be mild or moderate in severity as per the investigators.