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VX-661 plus ivacaftor combination

Vertex Pharmaceuticals Incorporated · Phase 3 active Small molecule

VX-661 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, and ivacaftor is a CFTR potentiator.

VX-661 is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, and ivacaftor is a CFTR potentiator. Used for Cystic fibrosis in patients with one or two copies of the F508del mutation.

At a glance

Generic nameVX-661 plus ivacaftor combination
SponsorVertex Pharmaceuticals Incorporated
Drug classCFTR modulator
TargetCFTR
ModalitySmall molecule
Therapeutic areaRespiratory
PhasePhase 3

Mechanism of action

The combination of VX-661 and ivacaftor works by correcting and potentiating the function of the CFTR protein, which is responsible for transporting chloride ions across cell membranes in the lungs, liver, pancreas, and intestine. This helps to improve the function of these organs and reduce the severity of cystic fibrosis symptoms.

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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