Tezacaftor is a small molecule drug that acts as a CFTR corrector, primarily used in the treatment of Cystic Fibrosis (CF). It works by addressing the defective CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein, which is responsible for the disease's symptoms. The drug is often used in combination with other CFTR modulators to improve its efficacy.
