Sacrosidase
| Evidence Level: L5 | Predicted Indications: 2 |
Table of Contents
Sacrosidase: From Congenital Sucrase-Isomaltase Deficiency to Cystinosis
One-Sentence Summary
Sacrosidase is an orally administered yeast-derived sucrase enzyme, originally used to treat congenital sucrase-isomaltase deficiency. The TxGNN model predicts it may be effective for Cystinosis, but currently there are 0 clinical trials and 0 publications supporting this direction, so the prediction rests on computation alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Congenital sucrase-isomaltase deficiency |
| Predicted New Indication | Cystinosis |
| TxGNN Prediction Score | 99.44% |
| Evidence Level | L5 |
| US Market Status | Cancelled only |
| Number of NDAs | 1 (BLA 020772, cancelled) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available. Based on known information, sacrosidase is an oral enzyme replacement that hydrolyzes dietary sucrose in the gastrointestinal lumen and is not systemically absorbed. Its efficacy in congenital sucrase-isomaltase deficiency rests on this local digestive action.
Cystinosis is a lysosomal storage disorder caused by CTNS mutations. These impair the cystinosin transporter and lead to cystine accumulation inside lysosomes. The standard of care is cysteamine, which depletes cystine. A luminal digestive enzyme has no plausible effect on lysosomal cystine transport, so no credible mechanistic link was identified.
The high score (0.994) is most likely explained by both conditions sitting in a similar “inherited metabolic disorder” neighborhood of the knowledge graph, not by a true biological relationship. It should be treated as a computational hypothesis only.
Another prediction from the same analysis: familial apolipoprotein C-II deficiency (score 99.05%) also lacks any mechanistic link, clinical trials or literature. It involves a defect in lipoprotein lipase activation, which a gut-luminal sucrase does not influence.
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 |
|---|---|---|---|
| BLA 020772 | Sucraid (QOL Medical, LLC) | Solution | Status: cancelled |
Safety Considerations
Please refer to the package insert for safety information.
No drug-drug interaction records were found in the data compiled for this report.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction has no clinical trial or literature support (L5), and no plausible mechanism links a non-absorbed gut enzyme to lysosomal cystine transport. The only US license is cancelled, so there is also no commercial basis for development.
To proceed, the following is needed:
- Mechanism of action data (e.g., from DrugBank) to allow a proper mechanistic assessment
- Package insert warnings and contraindications from the FDA, which are required before any safety screening
- Preclinical evidence of any systemic or lysosomal effect of sacrosidase relevant to cystinosis
- Clarification of the regulatory and supply status, given that the only US license is cancelled
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.