Tezacaftor

BeschreibungTezacaftor (VX-661) is an oral medication for cystic fibrosis (CF) developed by Vertex Pharmaceuticals. In a combination therapy formulation called Symdeko (tezacaftor plus ivacaftor), it is approved in the U.S. and Canada for CF patients, ages 12 and older, who have two copies of the F508del mutation in the CFTR gene and one minimal function mutation. Symdeko is approved and marketed in the EU as Symkevi.Tezacaftor is not approved as a stand-alone treatment, but as part of a combination therapy.

VerwendenTezacaftor is used as a combination therapy with Ivacaftor for the treatment of patients with cystic fibrosis.

DefinitionVX-661 is an investigational compound that promotes the maturation of delta F508 mutants of the cystic fibrosis transmembrane conductance regulator (CFTR). Delta F508 CFTR represents a class of CFTR mutation that is characterized by impaired processing of misfolded CFTR proteins and reduced accumulation of the protein at the cell surface. VX-661 is intended to facilitate trafficking of CFTR to the epithelial cell membrane. It may be combined with the CFTR potentiator ivacaftor (Item No. 15145) to stimulate both CFTR accumulation and opening at the apical epithelial surface.