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Vertex Pharmaceuticals recently announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive review suggesting the approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor) and ivacaftor combination drug regimen label expansion for treatment All CF patients aged 6 to 11 years with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
.
If approved, more than 1,500 children in Europe will be eligible for a drug that can treat the underlying cause of the disease for the first time
In Europe, the combination of Kaftrio and Kalydeco (ivacaftor) has been approved before: it is used to treat CF patients ≥12 years of age with at least one F508del mutation in the CFTR gene
.
On a global scale, F508del mutation is the most common cause of CF
In the United States, this triple therapy (Kaftrio+Kalydeco regimen) has been approved by the FDA in October 2019 under the brand name Trikafta (elexacaftor/tezacaftor/ivacaftor+ivacaftor)
.
In June 2021, the US FDA approved the expansion of the use of Trikafta: to include CF patients 6-11 years old who have at least one F508del mutation in the CFTR gene or a specific mutation in the CFTR gene and respond to Trikafta treatment based on in vitro data
Currently, the Kaftrio+ivacaftor program (Trikafta) has had a huge impact on the lives of eligible CF patients aged 12 years and above
.
Clinical data show that in 6-11 years old CF patients, Kaftrio+ivacaftor regimen (Trikafta) treatment can significantly improve lung function, sweat chloride, respiratory symptoms, safety and tolerability compared with observations in patients 12 years and older The results are consistent
