CFTR — Drug Target

All drugs that target CFTR — marketed and clinical-stage. Includes 3 drug classes acting on this target.

Drug classes

CFTR corrector · CFTR modulator · CFTR potentiator

Marketed (1)

• Symdeko (Copackaged) · Vertex Pharms · Respiratory
  Tezacaftor and ivacaftor work together to increase the amount and function of CFTR protein at the cell surface, enhancing chloride transport.

Phase 3 pipeline (8)

• ARINA-1 · Renovion, Inc. · CFTR corrector · Respiratory
  ARINA-1 is a small molecule inhaled cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
• VX-661/Ivacaftor · Vertex Pharmaceuticals Incorporated · CFTR corrector · Respiratory
  VX-661/Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
• VX-661 plus ivacaftor combination · Vertex Pharmaceuticals Incorporated · CFTR modulator · Respiratory
  VX-661 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, and ivacaftor is a CFTR potentiator.
• VX-121/TEZ/D-IVA · Vertex Pharmaceuticals Incorporated · CFTR modulator · Pulmonology
  VX-121/TEZ/D-IVA is a triple combination of a cystic fibrosis transmembrane conductance regulator (CFTR) corrector (VX-121), a CFTR potentiator (tezacaftor), and a CFTR amplifier (ivacaftor).
• VX-445/TEZ/IVA · Vertex Pharmaceuticals Incorporated · CFTR modulator · Pulmonology
  VX-445/TEZ/IVA is a combination of three cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
• VX-809 · Vertex Pharmaceuticals Incorporated · CFTR corrector · Cystic Fibrosis
  VX-809 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
• TEZ/IVA · Vertex Pharmaceuticals Incorporated · CFTR modulator · Pulmonology
  TEZ/IVA is a combination of tezacaftor and ivacaftor, which work together to increase the function of the CFTR protein in the lungs.
• VNZ/TEZ/D-IVA · Vertex Pharmaceuticals Incorporated · CFTR modulator · Cystic Fibrosis
  VNZ/TEZ/D-IVA is a triple combination therapy consisting of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, a corrector, and a potentiator.

Phase 2 pipeline (5)

• CHF5633 · Chiesi Farmaceutici S.p.A. · CFTR corrector · Respiratory
  CHF5633 is a small molecule in development for the treatment of cystic fibrosis.
• Elexacaftor-tezacaftor-ivacaftor exposure · Jennifer Taylor-Cousar · CFTR potentiator · Respiratory
  CFTR potentiator
• Ivacaftor Exposed · Vertex Pharmaceuticals Incorporated · CFTR potentiator · Respiratory
  Potentiates CFTR chloride channel function
• CFTR modulators · Hospices Civils de Lyon · CFTR modulator · Respiratory
  CFTR modulators work by modifying the cystic fibrosis transmembrane conductance regulator protein to restore normal chloride ion transport across epithelial cell membranes.
• CHF 1535 Next DPI · Chiesi Farmaceutici S.p.A. · CFTR modulator · Respiratory
  CHF 1535 Next DPI is a dry powder inhaler for the treatment of cystic fibrosis.