Tables
- Table 1. Mechanism of action and targets of emerging cystic fibrosis transmembrane conductance regulator (CFTR) modulators
Mutation class Nature of defect Example mutations Pancreatic function# Modulator strategy Agents in development I Biosynthesis G542X Insufficient Suppressor
Suppressor and potentiator¶Ataluren II Trafficking F508del Insufficient Corrector
Corrector plus potentiatorVX-809 plus ivacaftor+
VX-661 plus ivacaftor+III Channel gating G551D Insufficient Potentiator Ivacaftor (FDA approved, G551D)
Other gating mutations?¶IV Channel conductance R117H Sufficient Potentiator?¶ No clinical data available V Low transcript levels 3849+10kb C->T Sufficient Potentiator?¶ No clinical data available FDA: US Food and Drug Administration. #: when in trans with a non-functional CFTR mutation; ¶: theoretical approach based on nature of defect and known drug mechanism; +: currently in phase II trials. Reproduced from [15] with permission from the publisher.
Vol 22 Issue 127
Table of Contents
The impact of personalised therapies on respiratory medicine
