Description
Tezacaftor (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.
Uses
Tezacaftor is used as a combination therapy with Ivacaftor for the treatment of patients with cystic fibrosis.
Definition
VX-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.
Biochem/physiol Actions
VX-661 is another cystic fibrosis transmembrane conductance regulator (CFTR) corrector in development for the treatment of cystic fibrosis.
in vitro
VX-661, is CFTR modulator that is potentially useful for treatment of cystic fibrosis. VX-661 corrects F508del-CFTR trafficking and increases F508del-CFTR protein activity in vitro.
storage
Store at -20°C , stored under nitrogen
References
[1] s. donaldson, j. pilewski, m. griese, q. dong, p.-s. lee, for the vx11–661-101 study group. ws7.3 vx-661, an investigational cftr corrector, in combination with ivacaftor, a cftr potentiator, in patients with cf and homozygous for the f508del-cftr mutation: interim analysis. journal of cystic fibrosis, volume 12, supplement 1, june 2013, page s14
