Vertex announces EMA validation for marketing authorization of KAFTRIO

Vertex Pharmaceuticals announced that the European Medicines Agency, EMA, has validated a Type II variation application to the Marketing Authorization for KAFTRIO 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 ages 2 and above who have a mutation in the cystic fibrosis transmembrane conductance regulator 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, which will issue an opinion to the European Commission regarding the potential approval of this license expansion. Data 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. The medicine was generally well tolerated, with safety data generally consistent with the established safety profile of KAFTRIO in combination with ivacaftor.