Ivacaftor is currently under clinical evaluation to assess its efficacy and safety in infants with cystic fibrosis (CF). CF is a life shortening, inherited condition that occurs due to a faulty gene called cystic fibrosis transmembrane conductance regulator (CFTR). When functioning properly, the CFTR gene should produce a protein which maintains a balance in salt and water that moves into and out of the lungs. If this protein is not produced or does not function typically, then a thick and sticky saliva, called mucus, builds up in multiple organs, including the lungs and causes lung infections. Repeated lung infections and inflammation can cause long term damage and complications in other organs, such as the pancreas. Symptoms include frequent respiratory infections, poor nutritional status and stunted growth. However, there are no approved pharmacological treatment options for this patient population.
Ivacaftor for cystic fibrosis in infants
Ivacaftor is currently under clinical evaluation to assess its efficacy and safety in infants with cystic fibrosis (CF). CF is a life shortening, inherited condition that occurs due to a faulty gene called cystic fibrosis transmembrane conductance regulator (CFTR). When functioning properly, the CFTR gene should produce a protein which maintains a balance in salt and water that moves into and out of the lungs.