Tezacaftor
| Evidence Level: L5 | Predicted Indications: 3 |
Table of Contents
Tezacaftor: From Cystic Fibrosis to HIV Infectious Disease
One-Sentence Summary
Tezacaftor is a CFTR corrector, originally used in combination therapies for cystic fibrosis. The TxGNN model predicts it may be effective for HIV infectious disease, but there are currently no clinical trials and no publications supporting this direction, so the prediction rests on the model alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Cystic fibrosis |
| Predicted New Indication | HIV infectious disease |
| TxGNN Prediction Score | 99.24% |
| Evidence Level | L5 |
| New Zealand Market Status | Cancelled only (no currently valid authorizations) |
| Number of Authorizations | 6 (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. Tezacaftor is part of fixed-dose combination therapies for cystic fibrosis (Symdeko, Trikafta, and Alyftrek), and its efficacy in cystic fibrosis is established. Its pharmacological target is listed as CFTR.
No mechanistic link between tezacaftor and HIV infection is supported by the available data. The high TxGNN score (0.992) is a computational prediction only, with no clinical or literature corroboration. Any biological rationale, for example involving protein folding, trafficking, or host-pathogen pathways, would be speculative and needs independent validation.
The other two top-ranked predictions are leprosy (score 99.14%) and multiple endocrine neoplasia (score 99.06%). Both are likewise model-only predictions with no trials or literature.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
New Zealand Market Information
All six authorizations are cancelled. Approved indication text is not available for any of them.
| Authorization Number | Product Name | Dosage Form | Status |
|---|---|---|---|
| 2726211 | Alyftrek | Tablet: vanzacaftor 10 mg / tezacaftor 50 mg / deutivacaftor 125 mg | Cancelled |
| 2726203 | Alyftrek | Tablet: vanzacaftor 4 mg / tezacaftor 20 mg / deutivacaftor 50 mg | Cancelled |
| 2726238 | Trikafta | Oral granules: elexacaftor 100 mg / tezacaftor 50 mg / ivacaftor 75 mg, plus ivacaftor 75 mg, sachets | Cancelled |
| 2726246 | Trikafta | Oral granules: elexacaftor 80 mg / tezacaftor 40 mg / ivacaftor 60 mg, plus ivacaftor 59.5 mg, sachets | Cancelled |
| 2611694 | Trikafta | Tablet: elexacaftor 100 mg / tezacaftor 50 mg / ivacaftor 75 mg, plus ivacaftor 150 mg | Cancelled |
A sixth authorization, 2649748 (Trikafta tablet: elexacaftor 50 mg / tezacaftor 25 mg / ivacaftor 37.5 mg, plus ivacaftor 75 mg), is also cancelled.
Safety Considerations
- Drug Interactions: The interaction query completed with one record, which is a pharmacological target association (tezacaftor acting on CFTR) rather than an interaction with another drug.
Please refer to the package insert for warnings and contraindications, as these were not available in the data compiled for this report.
Conclusion and Next Steps
Decision: Hold
Rationale: The HIV prediction is supported only by a model score, with no clinical trials, no literature, and no established mechanistic link. In addition, all New Zealand authorizations are cancelled and the package insert safety data are missing.
To proceed, the following is needed:
- Mechanism of action data (for example from DrugBank) to assess any plausible link to HIV
- Medsafe package insert warnings and contraindications for safety screening
- Preclinical or in vitro studies testing tezacaftor against HIV
- Clarification of the New Zealand regulatory status, since all authorizations are cancelled
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.