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

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

CFTR corrector

Pre-clinical Restore CFTR Function
Hypertonic Saline

Improves mucus clearance by increasing hydration

Therapeutic not approved by FDA or EMA Mucociliary Clearance

Enhances protein expression of CFTR

Pre-clinical Restore CFTR Function

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function

Non-steroidal anti-inflammatory drug and CFTR corrector

FDA-/EMA-approved for Other Purpose Anti-Inflammatory

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