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SUZ

Vertex Pharmaceuticals Incorporated · FDA-approved active Small molecule

SUZ is a CFTR potentiator that enhances the function of cystic fibrosis transmembrane conductance regulator protein to improve chloride transport in cystic fibrosis patients.

SUZ is a CFTR potentiator that enhances the function of cystic fibrosis transmembrane conductance regulator protein to improve chloride transport in cystic fibrosis patients. Used for Cystic fibrosis in patients with specific CFTR mutations responsive to potentiation.

At a glance

Generic nameSUZ
Also known asVX-548
SponsorVertex Pharmaceuticals Incorporated
Drug classCFTR potentiator
TargetCFTR (cystic fibrosis transmembrane conductance regulator)
ModalitySmall molecule
Therapeutic areaPulmonary/Respiratory
PhaseFDA-approved

Mechanism of action

SUZ works by binding to and potentiating the CFTR protein, increasing its channel-opening probability and duration, thereby enhancing chloride ion transport across epithelial cell membranes. This mechanism helps restore defective ion transport in cystic fibrosis patients, particularly those with certain CFTR mutations. The drug is designed to improve lung function and reduce disease progression in eligible CF populations.

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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