Desmopressin
| Evidence Level: L4 | Predicted Indications: 7 |
Table of Contents
Desmopressin: From Its Marketed Uses to Congenital Prothrombin Deficiency
One-Sentence Summary
Desmopressin is a synthetic vasopressin analogue (V2 receptor agonist) already marketed in the United States in several dosage forms. The TxGNN model predicts it may be effective for congenital prothrombin deficiency, but there is 0 directly relevant clinical trials and 0 publications on desmopressin for this disease. The only related items found concern other bleeding disorders, so the prediction rests on the model score alone.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Congenital prothrombin deficiency |
| TxGNN Prediction Score | 99.70% |
| Evidence Level | L4 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 20 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the record. From general pharmacology, desmopressin activates the V2 receptor and triggers release of von Willebrand factor (VWF) and factor VIII from endothelial stores. This shortens bleeding time. It is used clinically in bleeding conditions such as mild hemophilia A and certain subtypes of von Willebrand disease.
The link to congenital prothrombin deficiency is weak. Prothrombin (factor II) is a different clotting factor, and desmopressin has no known effect on prothrombin levels. The prediction seems to come from the general “bleeding disorder” context in the knowledge graph rather than from a shared mechanism. The high score (0.997) is therefore not backed by direct clinical evidence.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT04567511 | Phase 4 | Recruiting | 20 | Single-arm study of emicizumab (Hemlibra) in mild hemophilia A. It tests a different drug in a different disease, so it gives no direct evidence (relevance grade C). |
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 21115138 | 2011 | Review | Autoimmunity Reviews | Acquired hemophilia A: diagnosis, causes, clinical spectrum and treatment options. It is a different disease and not specific to desmopressin. |
| 7684674 | 1993 | Review | Drugs | Treatment options for common inherited bleeding disorders such as hemophilia A and von Willebrand disease. It is not specific to prothrombin deficiency. |
| 2607619 | 1989 | Case report | Rinsho Ketsueki | DDAVP given to a patient with congenital combined factor V and factor VIII deficiency. This is not a prothrombin deficiency. |
| 1942544 | 1991 | Case report | Rinsho Ketsueki | Cesarean section managed with factor VIII concentrate in combined factor V and VIII deficiency. It is not desmopressin-specific. |
US Market Information
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| ANDA074830 | Desmopressin Acetate | Solution | Oceanside Pharmaceuticals |
| NDA018938 | Desmopressin Acetate | Solution | Nordic Pharma, Inc. |
| NDA019955 | DDAVP | Tablet | Ferring Pharmaceuticals Inc. |
| ANDA201831 | Desmopressin Acetate | Tablet | Glenmark Pharmaceuticals Inc., USA |
| NDA019955 | Desmopressin Acetate | Tablet | Nordic Pharma, Inc. |
Available routes: oral (tablet), injectable (injection, solution), and other (solution).
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: No study tests desmopressin in congenital prothrombin deficiency. The only trial found involves another drug, and the literature covers other bleeding disorders. The mechanism (VWF and factor VIII release) does not address prothrombin. The other six predicted indications are also not ready to advance: three are graded Hold and two Research Question, and the seventh, “flood factor deficiency,” looks like a naming artifact. The exception is primary release disorder of platelets, which is graded L3 and Research Question.
To proceed, the following is needed:
- FDA package insert warnings and contraindications (a blocking gap for safety screening)
- Mechanism of action data from DrugBank
- Direct evidence on desmopressin in prothrombin deficiency, such as case series or mechanistic data
- A clear rationale for why desmopressin would raise or substitute for prothrombin activity
- Route compatibility assessment for the intended use
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.