VX-522 + ivacaftor

VX-522 + ivacaftor is a combination therapy under investigation for the treatment of cystic fibrosis (CF) in adults who have CFTR mutations unresponsive to existing CFTR modulator drugs. VX-522 is an investigational mRNA-based therapy designed to deliver functional CFTR mRNA into airway cells, enabling the production of a working CFTR protein where none is present. Ivacaftor is a CFTR potentiator that increases the probability of the CFTR chloride channel being open, thereby improving chloride transport at the cell surface. This combination seeks to address the underlying protein defect in CF patients with minimal or no functional CFTR, with VX-522 providing the genetic instructions for protein synthesis and ivacaftor enhancing the function of any expressed CFTR protein[1][2][3].

Other names

IVAivacaftor