Press release
Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO® in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations
-Application to add ~200 non-F508del CFTR mutations to the KAFTRIO® license- -If approved, ~2,800 people with cystic fibrosis in the European Union ages 2
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n -Application to add ~200 non-F508del CFTR mutations to the KAFTRIO® license-\n\n\n-If approved, ~2,800 people with cystic fibrosis in the European Union ages 2 and above could receive a medicine that treats the underlying cause of their disease for the first time-\n\n\n LONDON--(BUSINESS WIRE)--\nVertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency (EMA) has validated a Type II variation application to the Marketing Authorization for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor. The application is for expansion of the approved indication for KAFTRIO® in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis (CF) ages 2 and above who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical and/or in vitro data, including the N1303K mutation. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval of this license expansion.\n\n\nData to support this submission includes the results of a Phase 3, randomized, placebo-controlled clinical study in people with rare non-F508del KAFTRIO®-responsive CFTR mutations. This study met its primary endpoint and showed that KAFTRIO® in combination with ivacaftor resulted in rapid, statistically significant, and clinically meaningful improvements in lung function compared to placebo (9.2 percentage point increase in ppFEV1; P","length":2255,"tagName":"div"}]