Summary
Crofelemer 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. No registered clinical trial in this database tests Crofelemer specifically in Cystic Fibrosis. The evidence below comes from published literature only, so any clinical signal has not yet been tested prospectively against this condition in a registered study. The literature layer holds 2 publications for this pair: 1 Cochrane review and 1 clinical trial publication. Publication years run from 2014 to 2017. 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 | 27.0 (trials 0.0, literature 7.0, tier 15, approved bonus 5.0) |
| Registered trials | 0 total: 0 recruiting, 0 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 2 (1 Cochrane review, 1 clinical trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | inhibitor; inhibitor |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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
No registered trial links Crofelemer to Cystic Fibrosis in the current OSMF trial extract.
Published literature
- Clinical trial publication HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based RegimensGao JJ, Tan M, Pohlmann PR et al. · Clinical breast cancer · 2017 · PMID 27692565
- Cochrane review Crofelemer: In HIV Associated Diarrhea and Secretory Diarrhea - A Patent PerspectiveBiswal S · Recent patents on anti-infective drug discovery · 2014 · PMID 25851117
Mechanism and notes
Recorded mechanism or class: inhibitor; inhibitor.
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 Crofelemer approved for Cystic Fibrosis?
Crofelemer 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 Crofelemer in clinical trials for Cystic Fibrosis?
No registered clinical trial in this database tests Crofelemer specifically in Cystic Fibrosis. The evidence below comes from published literature only, so any clinical signal has not yet been tested prospectively against this condition in a registered study.
What does the evidence show for Crofelemer in Cystic Fibrosis?
Synthesis-level literature links Crofelemer to Cystic Fibrosis, but no registered trial in this database tests the pair, so the evidence is observational, pooled from other indications, or pre-dates registry practice. 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.