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 -November 27, 2023 at 08:54 am EST
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
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 (9.2 percentage point increase in ppFEV1; P
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