The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) expressed a positive attitude toward the label extension of ivacaftor/tezacaftor/elexacaftor (Kaftrio®) in combination with ivacaftor to treat children with cystic fibrosis (CF) ages 2 to 5 years who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, Vertex Pharmaceuticals announced.

“Kaftrio has demonstrated unprecedented clinical benefit for eligible people living with CF,” Nia Tatsis, PhD, executive vice president, chief regulatory and quality officer at Vertex stated.

“Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for Kaftrio to patients as young as 2 years.”

The medication combination has already been approved for the treatment of people with CF ages 6 years and older who have at least one copy of the F508del mutation in the CFTR gene in the European Union.

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A mixed regimen of ivacaftor/tezacaftor/elexacaftor and ivacaftor may cause headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, and rash, as well as serious adverse events such as liver damage and worsening of liver function, serious allergic reactions, and abnormality of the eye lens (cataract).


Vertex receives CHMP positive opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for children with cystic fibrosis ages 2 through 5. News release. Vertex Pharmaceuticals Inc.; September 15, 2023.