Vertex withdraws EU filings to expand Kaftrio/Kalydeco to younger children
核心洞察
Vertex Pharmaceuticals withdrew EU applications to extend Kaftrio and Kalydeco combination treatment to cystic fibrosis (搜索) patients aged one year and older.
The EMA published notice on September 18 that Vertex Pharmaceuticals (Ireland) Limited pulled the Kalydeco (ivacaftor) extension, with the withdrawal dated September 1, 2026.
A parallel Kaftrio (ivacaftor/tezacaftor/elexacaftor) granule application for the same age extension was also withdrawn after EMA preliminary feedback requested more information.
Vertex Pharmaceuticals has withdrawn EU applications seeking to extend Kaftrio and Kalydeco combination treatment to children with cystic fibrosis (搜索) aged one year and older. The European Medicines Agency (搜索) published notice on September 18 that Vertex Pharmaceuticals (Ireland) Limited had withdrawn its application to extend Kalydeco (ivacaftor), used in combination with a medicine containing ivacaftor/tezacaftor/elexacaftor, to children aged one year and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 CFTR (搜索) 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 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, and Vertex had not responded to those questions at the time of withdrawal. In its formal withdrawal letter, Vertex said it pulled the application because the EMA's preliminary feedback indicated additional information would be required to support the indication extension. Vertex had submitted 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 no formal recommendation had been issued at the time of withdrawal.
Kalydeco has held EU authorization since July 2012 for use as monotherapy in CF patients aged one month and older weighing at least 3 kg with eligible CFTR (搜索) mutations, and in combination regimens for patients aged two years and above. Kaftrio, authorized in the EU since August 2020, is approved for patients aged two years and older with at least one non-class I CFTR mutation. The EMA confirmed neither withdrawal affects these existing authorizations or patients currently enrolled in clinical trials. The withdrawals leave the Kaftrio/Kalydeco combination approved in the EU from age 2 years, delaying Vertex's effort to extend triple-combination CFTR modulator treatment into younger children.