Marketing authorisation without orphan designation https://www.ema.europa.eu/en/medicines/human/EPAR/orkambi Europe R07AX30 ORKAMBI


                
                
                
                  Ingredient/substance
                
                
                  Synthetic / extractive chemistry
                
                Lumacaftor / Ivacaftor


      
      
      2015-11-19 00:00:00.0
      EU/1/15/1059
      
        
          
          VERTEX PHARMACEUTICALS (IRELAND) LIMITED
          
            
              
                Eta
                Marketing authorisation holder
              
            
          
        
      
      
        
          586
          Cystic fibrosis
        
      
      Orkambi tablets are indicated for the treatment of <b>cystic fibrosis</b> (<b>CF</b>) in patients aged 6 years and older who are homozygous for the <i>F508del</i> mutation in the <i>CFTR</i> gene.<br> Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.