Filter the drugs by the phase of research, how they help and the responsive mutations.
Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene. Browse the
database of
drugs treating specific mutations compiled by the AIM tool.
| Drug | Marketed as | Description | Phase of research | How it helps |
|---|---|---|---|---|
| Ataluren | Translarna™ | Suppresses premature stop mutation |
EMA-approved for Other Purpose | Restore CFTR Function |
| ETX001 | TMEM16A potentiator |
Pre-clinical | Mucociliary Clearance | |
| Elexacaftor | Trikafta™ | CFTR corrector |
FDA-approved for Cystic Fibrosis | Restore CFTR Function |
| G418 | Aminoglycoside antibiotic capable of translational readthrough |
Pre-clinical | Restore CFTR Function | |
| Gentamicin | Aminoglycoside antibiotic capable of translational readthrough |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Ivacaftor | Kalydeco® (ivacaftor), Orkambi® (ivacaftor/lumacaftor), Symdeko® (ivacaftor/tezacaftor), Symkevi® (ivacaftor/tezacaftor), Trikafta™(elexacaftor/tezacaftor/ivacaftor) | CFTR potentiator |
FDA-/EMA-approved for Cystic Fibrosis | Restore CFTR Function |
| Tezacaftor | Symdeko® (ivacaftor/tezacaftor), Symkevi® (ivacaftor/tezacaftor), Trikafta™(elexacaftor/tezacaftor/ivacaftor) | CFTR corrector |
FDA-/EMA-approved for Cystic Fibrosis | Restore CFTR Function |
| VX-152 | CFTR corrector |
Phase Two Clinical Trial | Restore CFTR Function | |
| VX-440 | CFTR corrector |
Phase Two Clinical Trial | Restore CFTR Function | |
| VX-659 | CFTR corrector |
Phase Two Clinical Trial | Restore CFTR Function |
