Find drugs for your specific mutation

Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene. Each person with cystic fibrosis has two CFTR mutations – they can be the same or different. Enter your mutations to find the drugs that could help. The order of your mutations is not important. If you know only one of your mutations, choose “Don't know” for your second mutation.


results for 2184delA / W1098X

Approved CFTR modulators

KALYDECO 

ivacaftor
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO.

ORKAMBI

lumacaftor/ivacaftor
ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should not be used in patients other than those who have two copies of the F508del mutation in their CFTR gene.

SYMDEKO

tezacaftor/ivacaftor and ivacaftor
SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO.

TRIKAFTA

Elexacaftor + tezacaftor + ivacaftor
TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one F508del mutation in the CFTR gene. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one F508del mutation.
  • 30
    Total number of drugs
  • 16
    Number of FDA- or EMA-approved drugs
  • 12
    Number of drugs in development
  • 2
    Number of dietary supplements

Treats - 2184delA / W1098X

Drug Phase of research Number of sources Average impact factor
Amlexanox FDA-approved for Other Purpose 1 4.32

Treats - General effect

Drug Phase of research Number of sources Average impact factor
ALX-009 Phase One Clinical Trial 1 5.11
AquADEKs® Accessible Dietary Supplement 3 7.22
Mannitol EMA-approved for Cystic Fibrosis 3 8.79
Levofloxacin FDA-/EMA-approved for Cystic Fibrosis 3 6.55
Nitric oxide Phase Two Clinical Trial 1 7.01
OligoG Phase Two Clinical Trial 2 3.41
Pancrelipase enzyme FDA-approved for Cystic Fibrosis 1 4.73
Pancrelipase enzyme FDA-approved for Cystic Fibrosis 1 3.19
Pancrelipase enzyme FDA-approved for Cystic Fibrosis 1 13.77
Pancrelipase enzyme FDA-approved for Cystic Fibrosis 3 3.20
pGM169/GL67A Phase Two Clinical Trial 1 4.29
Polymyxin B FDA-/EMA-approved for Other Purpose 1 6.27
Recombinant human deoxyribonuclease I FDA-/EMA-approved for Cystic Fibrosis 9 5.53
SPX-101 Phase Two Clinical Trial 2 9.77
Tobramycin FDA-/EMA-approved for Cystic Fibrosis 2 3.19
Vancomycin Phase Three Clinical Trial 1 2.80
ETX001 Pre-clinical 1 16.49
Acebilustat Phase Two Clinical Trial 1 1.95
Alpha 1 Anti-Trypsin FDA-approved for Other Purpose 2 3.66
Amikacin FDA-approved for Cystic Fibrosis 2 N/A
Azithromycin FDA-approved for Cystic Fibrosis 4 18.51
Aztreonam FDA-/EMA-approved for Cystic Fibrosis 6 5.76
POL6014 Phase One Clinical Trial 1 4.29
Gallium Phase Two Clinical Trial 2 17.16
Docosahexaenoic Acid Accessible Dietary Supplement 2 3.25
KB001 Phase Two Clinical Trial 2 3.86
Hypertonic Saline Therapeutic not approved by FDA or EMA 6 22.30
Ibuprofen FDA-/EMA-approved for Other Purpose 4 5.79
Lipase FDA-approved for Cystic Fibrosis 1 3.02

Does not treat - 2184delA / W1098X

Drug Phase of research Number of sources Average impact factor