Summary
Deutivacaftor has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cystic Fibrosis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cystic Fibrosis. ClinicalTrials.gov lists 8 registered trials linking Deutivacaftor to Cystic Fibrosis: 3 are currently recruiting; 2 are active or not yet recruiting; 3 have completed. The most advanced is Phase 3 (NCT05444257). It plans or enrolled 822 participants. Registration activity spans 2018 to 2026. The literature layer holds 7 publications for this pair: 2 Cochrane reviews, 1 meta-analysis and 4 clinical trial publications. Publication years run from 2023 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Cystic Fibrosis ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Progressive lung function decline; no spontaneous remission; historically median survival 30 years; now >50 years with CFTR modulators for eligible genotypes.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 72.0 (trials 30.0, literature 22.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 3 recruiting, 2 active / not yet recruiting, 3 completed, 0 other |
| Linked publications | 7 (4 clinical trial publications, 2 Cochrane reviews, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | activator; activator |
| Data sources | DGIdb |
| Linked via biomarker / target | CFTR |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT05444257 | A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy 2022 | active, not recruiting | Phase 3 | 822 |
| NCT05033080 | A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) 2021 | completed | Phase 3 | 435 |
| NCT05076149 | A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation 2021 | completed | Phase 3 | 597 |
| NCT05844449 | Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older 2023 | enrolling by invitation | Phase 3 | 174 |
| NCT06154447 | Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis 2023 | active, not recruiting | Phase 1 | 165 |
| NCT07349394 | Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants 2026 | completed | Phase 1 | 18 |
| NCT04732910 | Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators 2018 | recruiting | Not applicable | 500 |
| NCT06683092 | Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL) 2025 | recruiting | Not applicable | 200 |
Published literature
- Clinical trial publication Improvements in health-related quality of life in people with cystic fibrosis ≥6 years of age treated with vanzacaftor/tezacaftor/deutivacaftorWainwright CE, Thorat T, Conner S et al. · Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026 · PMID 41350125
- Clinical trial publication Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trialHoppe JE, Kasi AS, Pittman JE et al. · The Lancet. Respiratory medicine · 2025 · PMID 39756425
- Clinical trial publication Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trialsKeating C, Yonker LM, Vermeulen F et al. · The Lancet. Respiratory medicine · 2025 · PMID 39756424
- Cochrane review Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysisSafeer V S M, Behl S, Vaidya PC et al. · EClinicalMedicine · 2025 · PMID 41377908
- Meta-analysis Comparative Efficacy of CFTR Modulators: A Network Meta-analysisIftikhar IH, Rao ST, Nadama R et al. · Lung · 2025 · PMID 40102290
- Clinical trial publication Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trialsUluer AZ, MacGregor G, Azevedo P et al. · The Lancet. Respiratory medicine · 2023 · PMID 36842446
- Cochrane review Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)Heneghan M, Southern KW, Murphy J et al. · The Cochrane database of systematic reviews · 2023 · PMID 37983082
Mechanism and notes
Recorded mechanism or class: activator; activator.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CFTR. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Deutivacaftor approved for Cystic Fibrosis?
Deutivacaftor has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cystic Fibrosis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cystic Fibrosis. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Deutivacaftor in clinical trials for Cystic Fibrosis?
ClinicalTrials.gov lists 8 registered trials linking Deutivacaftor to Cystic Fibrosis: 3 are currently recruiting; 2 are active or not yet recruiting; 3 have completed. The most advanced is Phase 3 (NCT05444257). It plans or enrolled 822 participants. Registration activity spans 2018 to 2026.
What does the evidence show for Deutivacaftor in Cystic Fibrosis?
Deutivacaftor has both completed registered trials and synthesis-level publications linked to Cystic Fibrosis. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CFTR. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.