EMA and MHRA validate regulatory applications for new use of Kaftrio

US biotech Vertex Pharmaceuticals (Nasdaq: VRTX) says that the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) have validated the post-marketing applications for an expanded indication of Kaftrio (ivacaftor/tezacaftor/elexacaftor) used in combination with ivacaftor to include patients ages six years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Trade-named Trikafta in the USA, Kaftrio is Vertex’ newest cystic fibrosis drug, which treats 90% of patients with the lung disease, and its sales surged 123% to $1.19 billion in the first quarter of this year.

If approved, this will extend the indication for the triple combination therapy to children ages six through 11 years old in European Union countries, Great Britain and Northern Ireland. The applications are supported by positive results from a global Phase III open-label study over 24 weeks that evaluated the safety and efficacy of ivacaftor/tezacaftor/elexacaftor plus ivacaftor in 66 children ages six through 11 years old who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.