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 |
|---|---|---|---|---|
| Sildenafil | CFTR activator |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Sodium 4-phenylbutyrate | Induces CFTR function |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| TCP10L | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| TFG | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| TMA | CFTR corrector and potentiator |
Pre-clinical | Restore CFTR Function | |
| Tadalafil | CFTR activator |
FDA-/EMA-approved for Other Purpose | 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 |
| Thymosin α1 | ZADAXIN™ | Natural polypeptide reducing inflammation and increasing CFTR maturation, stability and activity |
Therapeutic not approved by FDA or EMA | Restore CFTR Function + Anti-Inflammatory |
| Tobramycin | TOBI® Podhaler™ | Aminoglycoside antibiotic capable of translational readthrough |
FDA-/EMA-approved for Cystic Fibrosis | Anti-Infective,Restore CFTR Function |
| VX-152 | CFTR corrector |
Phase Two Clinical Trial | Restore CFTR Function |
