EC expands indication for Vertex' Kaftrio

The European Commission has granted approval for the label expansion of Kaftrio (ivacaftor/tezacaftor/elexacaftor; marketed in the USA as Trikaftra) in a combination regimen with ivacaftor for the treatment of children with cystic fibrosis (CF) ages two through five years old who have at least one F508del mutation in the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene, US biopharma Vertex Pharmaceuticals (Nasdaq: VRTX) announced today.

“In addition to data from clinical trials, 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 Dr Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer of Vertex.

Kaftrio/Trikaftra received strong international uptake in the third quarter of this year, with net product revenues up 6% to $929 million ex-USA. Sales were up 7% at $1.55 billion in the USA.