Vertex Pharmaceutical’s triple combination cystic fibrosis treatment, Kaftrio, is one step closer to coming to market after receiving a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
Vertex is seeking approval in Europe for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis in patients aged 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in their CFTR gene. Though the European Commission is not bound by the opinion of the CHMP, it usually seconds CHMP recommendations in its final decision for approval in Europe.
If approved in the EU, about 10,000 new patients with one minimal function mutation and one F508del mutation will beeligible to be treated with Kaftrio. Additionally, patients who have two F508del mutations and are presently eligible to be treated with one of Vertex’s other medicines — Kalydeco, Orkambi or Symdeko — will also be eligible for Kaftrio.
The CHMP recommendation was supported by positive data from two global phase III studies in CF patients. Both studies showed statistically significant improvements in lung function, which was the primary endpoint, and in all key secondary endpoints.