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 |
|---|---|---|---|---|
| Gentamicin | Aminoglycoside antibiotic capable of translational readthrough |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Hepatocyte growth factor | Enhances functional rescue of Phe508del-CFTR |
Pre-clinical | Restore CFTR Function | |
| HspA4 | CFTR corrector |
Pre-clinical | Restore CFTR Function | |
| Hypertonic Saline | Improves mucus clearance by increasing hydration |
Therapeutic not approved by FDA or EMA | Mucociliary Clearance | |
| IGF-1 | Enhances protein expression of CFTR |
Pre-clinical | Restore CFTR Function | |
| IPDMA | CFTR corrector and potentiator |
Pre-clinical | Restore CFTR Function | |
| IPEMA | CFTR corrector and potentiator |
Pre-clinical | Restore CFTR Function | |
| Ibuprofen | Non-steroidal anti-inflammatory drug and CFTR corrector |
FDA-/EMA-approved for Other Purpose | Anti-Inflammatory | |
| Imperatorin | Chinese medicinal herbs coumarin compound, F508del-CFTR activator |
Accessible Dietary Supplement | 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 |
