Pemigatinib
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Pemigatinib: From an Unspecified Original Indication to Multiple Endocrine Neoplasia
One-Sentence Summary
Pemigatinib is a selective FGFR1-3 inhibitor, and its original approved indication is not recorded in the data compiled for this report. The TxGNN model predicts it may be effective for multiple endocrine neoplasia, but there are 0 clinical trials and 0 publications supporting this direction. This prediction is based on the model score alone and is not supported by known biology.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not available (the Singapore licence records contain no indication text) |
| Predicted New Indication | Multiple endocrine neoplasia |
| TxGNN Prediction Score | 99.71% |
| Evidence Level | L5 |
| Singapore Market Status | ✓ Marketed |
| Number of Registrations | 3 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Pemigatinib is a selective inhibitor of FGFR1-3 (fibroblast growth factor receptors). Detailed mechanism of action data from DrugBank is not available for this report, and the original indication could not be confirmed from the Singapore licence records.
The link to the predicted indication is weak. Multiple endocrine neoplasia syndromes are driven mainly by MEN1 loss-of-function and RET gain-of-function mutations, not FGFR signalling. The high score (0.997) most likely reflects relationships within the knowledge graph rather than a documented biological mechanism. The prediction is therefore not supported by known biology.
Other predicted indications were also reviewed. Their scores are all similarly high (99.2%-99.5%), but none has clinical trial evidence:
- HER2-positive breast carcinoma is the only one with a biologically plausible rationale, because FGFR signalling can contribute to resistance to HER2-targeted therapy. The one linked publication is a general review of kinase inhibitors, not evidence specific to pemigatinib.
- Amenorrhoea, cytomegalovirus infection, amyotrophic lateral sclerosis (and related entries) and axial spondylometaphyseal dysplasia have no plausible FGFR-inhibition mechanism.
- Infectious bovine rhinotracheitis and malignant catarrh are veterinary diseases with no human relevance. They are likely artefacts of the knowledge graph.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available for the predicted indication, multiple endocrine neoplasia.
Singapore Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| SIN17044P | PEMAZYRE TABLETS 13.5 MG | Tablet | Not stated in licence record |
| SIN17042P | PEMAZYRE TABLETS 4.5 MG | Tablet | Not stated in licence record |
| SIN17043P | PEMAZYRE TABLETS 9 MG | Tablet | Not stated in licence record |
All three licences are valid single-ingredient products (oral tablets) from Lonza Tampa LLC.
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Targeted therapy (FGFR kinase inhibitor) |
| Myelosuppression Risk | Please refer to the package insert warnings and precautions |
| Emetogenicity Classification | Please refer to the package insert warnings and precautions |
| Monitoring Items | Please refer to the package insert warnings and precautions |
| Handling Protection | Please refer to the package insert warnings and precautions |
Safety Considerations
- Drug Interactions: The interaction check returned 50 interacting drugs, 20 of which are shown in the data compiled for this report. Several are rated Major, including:
- Aprepitant
- Dexamethasone
- Clarithromycin
- Cobicistat
- Lumacaftor
- Chloramphenicol
- Erythromycin
- Ketoconazole
- Fluconazole
Many of these are CYP3A-related, so co-medication should be reviewed carefully. Moderate interactions include cimetidine, clotrimazole, deferasirox, ivacaftor, zafirlukast, griseofulvin, elagolix and osilodrostat, among others.
Please refer to the package insert for warnings and contraindications. These were not available in the data compiled for this report.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction rests on the model score only (evidence level L5). There are no clinical trials or indication-specific publications, and the known biology of multiple endocrine neoplasia does not involve FGFR signalling. The package insert warnings and contraindications are also missing, so safety screening cannot proceed.
To proceed, the following is needed:
- The package insert (warnings, contraindications and approved indication) from the HSA website
- Mechanism of action data from DrugBank
- Preclinical or mechanistic evidence linking FGFR inhibition to multiple endocrine neoplasia
- If a repurposing direction is still wanted, a review of HER2-positive breast carcinoma, the only predicted indication with a plausible, though unvalidated, FGFR rationale
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.