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TEZ

Vertex Pharmaceuticals Incorporated · FDA-approved active Small molecule

TEZ (tezacaftor) is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly and traffic to the cell surface.

TEZ (tezacaftor) is a CFTR corrector that helps misfolded cystic fibrosis transmembrane conductance regulator (CFTR) protein fold correctly and traffic to the cell surface. Used for Cystic fibrosis in patients aged 6 years and older with at least one F508del mutation (in combination with ivacaftor), Cystic fibrosis in patients with specific CFTR mutations responsive to CFTR modulation.

At a glance

Generic nameTEZ
Also known asTezacaftor, VX-661, tezacaftor
SponsorVertex Pharmaceuticals Incorporated
Drug classCFTR corrector
TargetCFTR (cystic fibrosis transmembrane conductance regulator)
ModalitySmall molecule
Therapeutic areaPulmonary/Genetic Disorders
PhaseFDA-approved

Mechanism of action

TEZ works by binding to CFTR and assisting in proper protein folding, allowing the defective CFTR protein to reach the cell membrane where it can function. It is typically used in combination with ivacaftor (a CFTR potentiator) to enhance both the quantity and function of CFTR at the cell surface. This combination addresses the underlying defect in cystic fibrosis caused by certain CFTR mutations.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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