lumacaftor

Ligand id: 7481

Name: lumacaftor

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Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 5
Hydrogen bond donors 2
Rotatable bonds 6
Topological polar surface area 97.75
Molecular weight 452.12
XLogP 5.06
No. Lipinski's rules broken 1

Molecular properties generated using the CDK

No information available.
Summary of Clinical Use
In July 2015, a fixed-dose combination with ivacaftor (Orkambi®) was approved by the US FDA for the treatment of cystic fibrosis patients aged 12 years and older carrying the F508del mutation. FDA approval for Orkambi® was expanded in October 2016 to include children with cystic fibrosis aged 6 to 11 who are homozygous for the F508del mutation.
Mechanism Of Action and Pharmacodynamic Effects
VX-809 is reported to correct protein mis-folding in one of the two transmembrane domains of the F508del-CFTR [2]. This effects partial restoration of functional CFTR channels. Combination therapy with compounds with different structure-guided mechanisms by which they correct CFTR function (e.g. Ivacaftor) may provide additional benefit in the treatment of CF [1].