Deoxycholic Acid

Evidence Level: L5

Predicted Indications: 3

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Table of Contents

1. Deoxycholic Acid
2. Deoxycholic Acid: From an Injectable Cytolytic Agent (KYBELLA) to Autosomal Dominant Familial Hematuria-Retinal Arteriolar Tortuosity-Contractures Syndrome
   1. One-Sentence Summary
   2. Quick Overview
   3. Why is This Prediction Reasonable?
   4. Clinical Trial Evidence
   5. Literature Evidence
   6. US Market Information
   7. Safety Considerations
   8. Conclusion and Next Steps
   9. Disclaimer

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## Pharmacist Assessment Report

Deoxycholic Acid: From an Injectable Cytolytic Agent (KYBELLA) to Autosomal Dominant Familial Hematuria-Retinal Arteriolar Tortuosity-Contractures Syndrome

One-Sentence Summary

Deoxycholic acid is a secondary bile acid marketed in the US as an injectable cytolytic agent (KYBELLA). The TxGNN model predicts it may be effective for autosomal dominant familial hematuria-retinal arteriolar tortuosity-contractures syndrome, a rare monogenic vascular basement-membrane disorder. This prediction currently has 0 clinical trials and 0 publications behind it, so it rests on the model score alone.

Quick Overview

Item	Content
Original Indication	Not recorded in the data (marketed as KYBELLA, an injectable cytolytic product)
Predicted New Indication	Autosomal dominant familial hematuria-retinal arteriolar tortuosity-contractures syndrome
TxGNN Prediction Score	99.49%
Evidence Level	L5
US Market Status	✓ Marketed
Number of NDAs	1
Recommended Decision	Hold

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available. Deoxycholic acid is a secondary bile acid, and its marketed use is as a cytolytic agent given by injection.

The data support no mechanistic link between this drug and the predicted disease. The disease is a rare monogenic disorder of vascular basement membranes. The 99.49% score comes from graph-based prediction alone, with no trials, no literature and no known mechanism of action to back it. This prediction should be treated as a model output that needs independent validation.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

Currently no related literature available.

US Market Information

Authorization Number	Product Name	Dosage Form	Approved Indication
NDA206333	KYBELLA (Kythera Biopharmaceuticals Inc.)	Injection, solution	Not provided in the record

Safety Considerations

Please refer to the package insert for safety information. No drug-interaction records were found for this drug.

Conclusion and Next Steps

Decision: Hold

Rationale: The prediction is supported only by a model score, at evidence level L5. There is no clinical, literature or mechanistic support. The drug is a locally injected cytolytic agent, and the target disease is a monogenic vascular disorder with no plausible drug-specific link.

To proceed, the following is needed:

• Mechanism of action data from DrugBank
• Package insert warnings and contraindications, which are needed before any safety screening
• A drug-specific mechanistic rationale linking deoxycholic acid to the disease, or supporting preclinical data
• A review of the other two TxGNN predictions for this drug:
  • Diabetic nephropathy (score 99.32%) has L4 preclinical support. Most of it involves FXR/TGR5 signalling or other bile acids such as UDCA rather than deoxycholic acid itself. Its systemic relevance is doubtful because the marketed product is a local injection. It is classed as a research question, not a development candidate.
  • Brain small vessel disease 1 with or without ocular anomalies (score 99.49%) has only generic disease-background literature, with no drug-specific evidence.

    Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.

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