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 |
|---|---|---|---|---|
| Elexacaftor | Trikafta™ | CFTR corrector |
FDA-approved for Cystic Fibrosis | Restore CFTR Function |
| Gentamicin | Aminoglycoside antibiotic capable of translational readthrough |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function | |
| Ibuprofen | Non-steroidal anti-inflammatory drug and CFTR corrector |
FDA-/EMA-approved for Other Purpose | Anti-Inflammatory | |
| 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 |
| Levofloxacin | Quinsair® | Antibiotic |
FDA-/EMA-approved for Cystic Fibrosis | Anti-Infective |
| Lipase | RELiZORB® | Digestive enzyme cartridge |
FDA-approved for Cystic Fibrosis | Nutritional-GI-Other |
| Lumacaftor | Orkambi® (ivacaftor/lumacaftor) | CFTR corrector |
FDA-/EMA-approved for Cystic Fibrosis | Restore CFTR Function |
| Mannitol | Bronchitol® | Osmotic agent helping to moisten and thin the mucus in the airways |
EMA-approved for Cystic Fibrosis | Mucociliary Clearance |
| Miglustat | Zavesca® | CFTR corrector |
FDA-/EMA-approved for Other Purpose | Restore CFTR Function |
| Omalizumab | Xolair® | Treats CF-related allergic bronchopulmonary aspergillosis |
FDA-/EMA-approved for Other Purpose | Nutritional-GI-Other |
