The European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) have validated Vertex' applications to expand the scope of its cystic fibrosis (CF) treatment Kaftrio (ivacaftor/tezacaftor/elexacaftor).
The drugmaker is seeking approval for the drug's use in combination with ivacaftor in patients ages six years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
If approved, this will extend the indication for the triple combination therapy to children ages six through 11 years old in EU countries, Great Britain and Northern Ireland.
In children with CF, symptoms and organ damage including lung disease manifest very early in life.
“We are committed to working diligently with global regulators to expand the indication for our medicine such that younger people living with CF will also be able to access the triple combination therapy,” said Nia Tatsis, executive VP, chief regulatory and quality officer at Vertex.
Validation of the marketing applications “is an important milestone in broadening our access worldwide and we are pleased that we were able to file with the EMA and the MHRA in parallel”.