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

CFTR potentiator

Accessible Dietary Supplement Restore CFTR Function
Cysteamine Cystagon®

Proteostasis regulator, rescues the function of F508del-CFTR

FDA-/EMA-approved for Other Purpose Restore CFTR Function

CFTR corrector and potentiator

Pre-clinical Restore CFTR Function
Docosahexaenoic Acid

Lowers oxidative stress

Accessible Dietary Supplement Anti-Inflammatory

TMEM16A potentiator

Pre-clinical Mucociliary Clearance
Elexacaftor Trikafta™

CFTR corrector

FDA-approved for Cystic Fibrosis Restore CFTR Function

33-nucleotide antisense oligonucleotide designed to restore CFTR function

Phase One Clinical Trial Restore CFTR Function
Endothelin 1

CFTR corrector

Pre-clinical Restore CFTR Function
Epigallocatechin gallate

Flavonoid improving CFTR function

Accessible Dietary Supplement Restore CFTR Function

Proteasome inhibitor preventing premature CFTR breakdown

Pre-clinical Restore CFTR Function