Imiglucerase
| Evidence Level: L4 | Predicted Indications: 10 |
Table of Contents
Imiglucerase: From Gaucher Disease to Hurler Syndrome
One-Sentence Summary
Imiglucerase is a recombinant enzyme replacement therapy originally used to treat Gaucher disease. The TxGNN model predicts it may be effective for Hurler syndrome (MPS I), but there are currently 0 registered clinical trials and only 1 general review (not specific to this drug or disease), so the prediction rests almost entirely on computational similarity.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Gaucher disease |
| Predicted New Indication | Hurler syndrome |
| TxGNN Prediction Score | 99.52% |
| Evidence Level | L4 |
| Singapore Market Status | ✓ Marketed |
| Number of Registrations | 1 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not currently available. Based on known information, imiglucerase is a recombinant form of human glucocerebrosidase. It replaces the deficient enzyme in Gaucher disease, where glucosylceramide accumulates in lysosomes.
Hurler syndrome is a lysosomal storage disease caused by alpha-L-iduronidase deficiency. It therefore shares the broad context of lysosomal enzyme replacement with Gaucher disease. However, the link is categorical rather than mechanistic. Imiglucerase acts on glucosylceramide and has no known activity on glycosaminoglycan substrates. The high TxGNN score most likely reflects network proximity among lysosomal disorders. A specific enzyme therapy for MPS I (laronidase) already exists.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 21211680 | 2010 | Review | La Revue de médecine interne | General review of enzyme replacement therapy for lysosomal storage diseases. It describes the development of imiglucerase for Gaucher disease and ERT for other conditions, and offers no direct evidence for imiglucerase in Hurler syndrome. |
Singapore Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| SIN13842P | Cerezyme® (Imiglucerase) 400U Powder for solution for injection | Injection, powder, for solution | Not listed in the data compiled for this report |
Safety Considerations
- Drug Interactions: One interaction was identified, with miglustat (Moderate level).
Please refer to the package insert for other safety information, including warnings and contraindications.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction is supported only by a class-level link among lysosomal storage disorders. Imiglucerase has no substrate overlap with iduronidase, there are no clinical trials, and the sole publication is a general review. A purpose-specific therapy (laronidase) already exists for MPS I.
To proceed, the following is needed:
- Mechanism of action data, and a plausible biochemical rationale for activity against glycosaminoglycan substrates
- Preclinical evidence (for example, cell or animal models of MPS I) showing any effect of imiglucerase
- Package insert warnings and contraindications from the HSA
- A comparison against the existing standard of care for MPS I
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.