dirocaftor

Dirocaftor is an investigational small molecule drug developed as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. It is being studied for the treatment of cystic fibrosis (CF), particularly in patients with rare CFTR mutations. Dirocaftor works by enhancing the function of defective CFTR proteins at the cell surface, thereby improving chloride transport and potentially alleviating symptoms associated with CF such as impaired lung function and abnormal sweat chloride levels. In clinical trials, dirocaftor has been evaluated both as monotherapy and in combination with other CFTR modulators like nesolicaftor and posenacaftor, showing promising results in restoring near-normal CFTR activity[6][8].

Other names

dirocaftor