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

Targets a Pseudomonas aeruginosa virulence factor

Phase Two Clinical Trial Anti-Inflammatory
Levofloxacin Quinsair®


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

CFTR corrector

Pre-clinical Restore CFTR Function

Proteasome inhibitor preventing premature CFTR breakdown

Pre-clinical Restore CFTR Function

CFTR corrector

Pre-clinical Restore CFTR Function

CFTR corrector

Pre-clinical 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