First Bacterial Infection as an Alternative Clinical End Point for Regulatory Approval of Agents Targeting the Primary Cystic Fibrosis Defect

Considerable effort is being expended to discover and develop therapeutic agents capable of correcting or compensating for (ie to “cure”) the primary cystic fibrosis (CF) defect in the lung. Therapeutic strategies include improving the processing or functioning of mutant cystic fibrosis transmembrane regulator (CFTR) protein,¹ up- or down-regulating alternative ion channels,² and introducing exogenous DNA sequences capable of producing functional CFTR protein.³

CF, Cystic fibrosis, HRCT, High-resolution computerized tomography, NPD, Nasal potential difference, rhDNase, Recombinant human deoxyribonuclease, TIS, Tobramycin inhalation solution