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Drugs in development · updated 30 October 2026

Cystic fibrosis: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:586

As of 30 October 2026 there are 27 active clinical trials (recruiting or ongoing, phases I–III) on 30 drugs for cystic fibrosis. 14 are experimental, that is not yet approved in the United States.

27Active trials
14Experimental drugs
1Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
VX-121III2NCT05444257
4D-710II1NCT05248230
ANG003II1NCT07450547
ARCT-032II1NCT06747858
BI 3720931I–II1NCT06962852
BX004II1NCT06998043
NHS7108II1NCT07559175
RCT2100II1NCT06237335
SPL84II1NCT06429176
Xenon-129II1NCT02740868
VX-581I2NCT07813039
KB407I1NCT05504837
VX-272I1NCT07437105
BMD003Early I2NCT07485543

Sorted by most advanced phase and number of active trials.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
EmpagliflozinJardianceII–III1NCT06149793
MaralixibatLivmarli®II–III1NCT06413368
TenapanorXphozah®, Ibsrela®III1NCT06810167
DefinityPerfluoropropaneII1NCT02740868
DulaglutideTrulicity®II1NCT04731272
IvacaftorKalydeco®II1NCT06237335
LosartanArbli®, Cozaar®II1NCT06364176
MetforminRiomet Er®, Glumetza®II1NCT04530383
PertzyePancrelipase, CreonII1NCT07450547
TamoxifenSoltamox®, Nolvadex®II1NCT07289035
VerapamilVerelan Pm®, Covera-Hs®II1NCT07688070
ZenpepII1NCT07559175
DeutivacaftorI1NCT07437105
Gallium nitrateGanite®I1NCT04294043
TezacaftorI1NCT07437105

Sorted by most advanced phase and number of active trials. Showing 15 of 16 drugs.

All 30 drugs, with the details

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D003550 “Cystic Fibrosis”. Prevalence: Orphanet.

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