Vertex Pharmaceuticals (NASDAQ:VRTX) has obtained approval from the European Commission for the expanded labeling of Kaftrio (ivacaftor/tezacaftor/elexacaftor) plus ivacaftor for the treatment of children with cystic fibrosis aged between two and five years.
The therapy is indicated for children who have at least at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“In addition to clinical trial data, long-term and real-world data have demonstrated the significant clinical benefit of Kaftrio in eligible people living with CF, and today’s news means that young children across Europe can now benefit from this important medicine. said Carmen Bozic, MD, executive vice president of Medical Affairs and Global Drug Development and chief medical officer of Vertex.
As a result of existing reimbursement arrangements in Austria, Denmark, Ireland, Norway, Latvia and Sweden, eligible patients in these countries will have access to the expanded indication for Kaftrio in combination with ivacaftor shortly after EC approval.
