EMA to review Vertex MAA change request

The European Medicines Agency (EMA) has validated Vertex Pharmaceuticals' (Nasdaq: VRTX) Type II variation application to the Marketing Authorization for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor.

This application is for expansion of the approved indication for Kaftrio alongside ivacaftor to treat people with cystic fibrosis (CF) aged two and above who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical or in vitro data, including the N1303K mutation.

"We are dedicated to bringing treatments to all people with CF"The application will now be reviewed by the EMA’s Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval of this license expansion.