CHMP positive for Kaftrio for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation
Vertex Pharmaceuticals Incorporated announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“Kaftrio has demonstrated unprecedented clinical benefit for eligible people living with CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “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.”
In the European Union, Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is already 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.
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