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lumacaftor + ivacaftor

Development stage: Approved
Lead developer: Vertex
Modality: Small Molecules
Administration: Oral

Overview

**Lumacaftor + ivacaftor** is an oral fixed-dose cystic fibrosis transmembrane conductance regulator modulator combination developed by Vertex for cystic fibrosis in people homozygous for the F508del mutation in the *CFTR* gene. Lumacaftor is a CFTR corrector that improves the folding, processing, and trafficking of F508del-CFTR to the cell surface, while ivacaftor is a CFTR potentiator that increases the channel-open probability of CFTR protein at the cell membrane. The combination is marketed as **Orkambi** and is supplied as immediate-release tablets and oral granules.

Brand names: Orkambi
Other names: ivacaftor + lumacaftorlumacaftor/ivacaftorLUM/IVA

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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