Vertex Pharmaceuticals has withdrawn EU applications seeking to extend Kaftrio and Kalydeco combination treatment to children with cystic fibrosis aged one year and older, after preliminary EMA feedback indicated that additional information would be needed to support the expansion.
The European Medicines Agency (EMA) published notice on September 18 that Vertex Pharmaceuticals (Ireland) Limited had withdrawn its application to extend Kalydeco (ivacaftor) — used in combination with a medicine containing ivacaftor/tezacaftor/elexacaftor — to children aged one year and older with CF who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The withdrawal, dated September 1, 2026, came alongside a parallel pullback for Kaftrio (ivacaftor/tezacaftor/elexacaftor), which sought the same age extension in granule form.
According to the EMA's published questions-and-answers document, the agency had completed its initial evaluation and prepared a list of questions for Vertex when the company chose to withdraw. Vertex had not responded to those questions at the time of withdrawal. In its formal withdrawal letter, the company said it pulled the application because the EMA's preliminary feedback indicated that additional information would be required to support the indication extension.
To support the applications, Vertex had submitted available efficacy and safety data from older children and adults treated with the ivacaftor/tezacaftor/elexacaftor combination, supplemented by pharmacokinetic and safety data from a study in children aged one to two years. The EMA's preliminary position suggested that package was insufficient, though the agency had not issued a formal recommendation at the time of withdrawal.