Nilotinib
| Evidence Level: L4 | Predicted Indications: 1 |
Table of Contents
Nilotinib: From Chronic Myeloid Leukemia to Dermatofibrosarcoma Protuberans
One-Sentence Summary
Nilotinib is an oral tyrosine kinase inhibitor that targets BCR-ABL, KIT and PDGFR. It is generally known as a treatment for chronic myeloid leukemia, although the license records in this Evidence Pack do not list an indication. The TxGNN model predicts it may be effective for dermatofibrosarcoma protuberans (DFSP), with 0 clinical trials and 1 publication (a review) currently supporting this direction. This is a mechanism-based model prediction, not clinical proof.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not listed in the license records; generally known as chronic myeloid leukemia (background knowledge, not from the Evidence Pack) |
| Predicted New Indication | Dermatofibrosarcoma protuberans |
| TxGNN Prediction Score | 99.31% |
| Evidence Level | L4 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 20 (total license records, including generic ANDAs) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data for nilotinib is not available in the input data. Based on known information, nilotinib is a kinase inhibitor that acts on PDGFR, BCR-ABL and KIT, so it may be mechanistically applicable to DFSP.
Most DFSP tumors carry a COL1A1-PDGFB fusion, which drives constitutive PDGFRB signaling. Because nilotinib inhibits PDGFR, blocking this pathway is a plausible strategy. Imatinib is a related tyrosine kinase inhibitor and an established PDGFR-directed therapy for this disease, which supports a class-level rationale.
The high TxGNN score (99.31%) is consistent with this link. Drug-specific mechanistic detail could not be checked because the input data lacks the mechanism of action and original indications.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 29408302 | 2018 | Review | Pharmacological Research | Reviews the role of small molecule PDGFR inhibitors in neoplastic disorders. It covers the PDGF growth factor family and receptor signaling as a therapeutic target. The abstract was truncated in the input, so DFSP- or nilotinib-specific findings could not be confirmed. |
US Market Information
The license records contain no approved indication text. Dosage forms reported are oral capsule and oral tablet. The list below shows 4 unique authorizations; ANDA209651 appears twice in the data.
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| NDA218922 | Nilotinib | Capsule | Cipla USA Inc. |
| ANDA209651 | Nilotinib | Capsule | Camber Pharmaceuticals, Inc. |
| ANDA218544 | Nilotinib | Capsule | Novadoz Pharmaceuticals LLC |
| ANDA218532 | Nilotinib | Capsule | Torrent Pharmaceuticals Limited |
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Targeted therapy (tyrosine 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
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction is mechanistically plausible (PDGFR inhibition in COL1A1-PDGFB-driven DFSP, with imatinib as a class precedent). However, the only support is a high model score and one general review, with no registered clinical trials. Package insert safety data is also missing, which blocks safety screening.
To proceed, the following is needed:
- Package insert warnings and contraindications (a blocking gap), obtained from the FDA website
- Mechanism of action data, obtained from DrugBank
- Original approved indication text from the license records
- Clinical evidence specific to nilotinib in DFSP (case series, trials, and comparison with imatinib)
- A literature search for nilotinib-specific DFSP studies, since the current review’s abstract does not confirm any
- Confirmation of route compatibility (currently pending)
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.