Nystatin
| Evidence Level: L4 | Predicted Indications: 10 |
Table of Contents
Nystatin: From Candidiasis to Vulvovaginitis
One-Sentence Summary
Nystatin is a polyene antifungal originally used to treat cutaneous, oral and vulvovaginal candidiasis. The TxGNN model predicts it may be effective for vulvovaginitis, but this largely overlaps with its existing candidiasis use. Currently there are 0 clinical trials and about 20 publications (mostly reviews, with no nystatin-specific RCT) supporting this direction.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Cutaneous candidiasis, oral candidiasis, candidiasis, vulvovaginal candidiasis |
| Predicted New Indication | Vulvovaginitis |
| TxGNN Prediction Score | 99.92% |
| Evidence Level | L4 |
| US Market Status | Cancelled only (no currently valid licenses) |
| Number of NDAs | 12 (all ANDAs, all cancelled) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in the data compiled for this report. Based on general pharmacology, nystatin is a polyene antifungal that binds ergosterol in the fungal cell membrane, forming pores and causing leakage of cell contents.
Vulvovaginal candidiasis, already an original indication, is a major cause of vulvovaginitis. The prediction therefore largely overlaps with the existing indication rather than being a true repurposing. The mechanism applies only to fungal (candidal) vulvovaginitis. It would not be expected to work in bacterial, atrophic or other non-infectious forms.
The literature found is mostly reviews, clinical overviews and comparator papers. Reviews note that nystatin, introduced in the 1950s for vulvovaginal candidiasis, has been surpassed by imidazoles and triazoles as first-choice treatment. More recent reviews discuss nystatin as an alternative option in fluconazole-resistant disease.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 39771534 | 2024 | Review | Pharmaceutics | Management of fluconazole-resistant vulvovaginal candidiasis; alternatives include boric acid, nystatin, oteseconazole and ibrexafungerp |
| 25775428 | 2015 | Review | BMJ Clinical Evidence | Vulvovaginal candidiasis is the second most common cause of vaginitis; C. albicans accounts for 85–90% of cases |
| 21774671 | 2011 | Review | J Womens Health | Clinical evidence for boric acid in recurrent vulvovaginal candidiasis; non-albicans species are more azole-resistant |
| 20406393 | 2011 | Clinical study | Mycoses | Compared fluconazole and nystatin susceptibility with clinical outcome in 283 patients with complicated vulvovaginal candidosis |
| 10796183 | 2000 | Systematic review | Cochrane Database Syst Rev | Topical treatment for vaginal candidiasis in pregnancy |
| 18729533 | 2008 | Review | Drugs | Local versus oral treatment of vulvovaginal candidosis; both considered equally effective in uncomplicated disease |
| 1436934 | 1992 | Review | Obstet Gynecol Clin North Am | Topical antifungals; nystatin surpassed by imidazoles and triazoles as first choice |
| 36588489 | 2022 | Clinical study | Rom J Morphol Embryol | Clinical, microbiological and histopathological aspects of candida vaginitis before local nystatin treatment |
| 41745294 | 2026 | Review | J Fungi (Basel) | Current and emerging antifungal agents for vulvovaginal candidiasis; azoles and polyenes remain the cornerstone |
| 16047929 | 2005 | Clinical study | Ceska Gynekol | Treatment of mixed vulvovaginal infections with combined nifuratel and nystatin vaginal products |
US Market Information
All 12 authorizations are listed as cancelled, and no approved-indication text was available. Up to 5 are shown below.
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ANDA203621 | Nystatin | Suspension | Not available |
| ANDA062506 | Nystatin | Tablet, film coated | Not available |
| ANDA062225 | Nystatin | Cream | Not available |
| ANDA207767 | Nystatin | Ointment | Not available |
| ANDA208581 | Nystatin | Powder | Not available |
Safety Considerations
- Disease associations from the knowledge graph (not label text): adrenocortical insufficiency, Addison disease, skin atrophy, telangiectasis, capillary disease, hereditary hemorrhagic telangiectasia. These should be verified against the official label.
- Drug Interactions: 100 interactions were found, all of unknown severity level. Examples include cyclosporine, fluconazole, erythromycin, azithromycin, bortezomib, tramadol, lorazepam and pantoprazole.
Please refer to the package insert for warnings and contraindications.
Conclusion and Next Steps
Decision: Hold
Rationale: The predicted indication largely overlaps with nystatin’s existing candidiasis use, so it is not a genuine new repurposing opportunity. Evidence consists of reviews with no registered trials or nystatin-specific RCT. Nystatin has also been displaced by azoles as first-line therapy, and all US licenses are cancelled. The other nine predictions (e.g., orbital disease, cystic teratoma, atrophic vaginitis) have no plausible mechanism or supporting data and should also be held.
To proceed, the following is needed:
- FDA package insert warnings and contraindications
- Detailed mechanism of action data
- Nystatin-specific controlled trials in vulvovaginitis, ideally against azoles or in fluconazole-resistant disease
- Clarification of current US market availability, since all licenses are cancelled
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.