# Ponatinib

Source: https://onco.cc/drugs/ponatinib/  
OnCo record `ponatinib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Ponatinib is the only BCR::ABL1 inhibitor that covers the T315I resistance mutation. In 2024 it became the preferred pill for newly diagnosed Ph-positive ALL.

## Summary

PhALLCON (n=245): ponatinib vs imatinib with reduced-intensity chemotherapy in newly diagnosed Ph+ ALL, MRD-negative CR at end of induction 34.4% vs 16.7%; accelerated approval March 2024. Also approved in CML (T315I and resistant disease) since 2012, with a 2013 temporary market suspension for arterial occlusive events that led to dose-reduction strategies (OPTIC). Ponatinib + blinatumomab chemotherapy-free regimens (MD Anderson) report near-universal molecular remission without transplant.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Iclusig
- Modality: Small-molecule kinase inhibitor (pan-BCR::ABL1 incl. T315I)
- Mechanism: Type II TKI binding inactive ABL1 with a carbon-carbon triple bond that accommodates the T315I gatekeeper; also inhibits FGFR, VEGFR, SRC.
- Approvals: US 2012: CML and Ph+ ALL resistant to prior TKIs or with T315I; US 2024: Newly diagnosed Ph+ ALL with chemotherapy (accelerated)
- Dosing: Oral; Ph+ ALL: 30 mg daily with chemotherapy, reduced to 15 mg once MRD-negative CR is achieved; CML: 45 mg daily reducing to 15 mg on response (OPTIC)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Ponatinib
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ponatinib

## Connected records

- biomarkers: [BCR::ABL1 T315I](https://onco.cc/biomarkers/bcr-abl1-t315i/), [BCR::ABL1 transcript (Philadelphia chromosome, quantitative PCR)](https://onco.cc/biomarkers/bcr-abl1-transcript/)
- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Chronic myeloid leukaemia (CML)](https://onco.cc/cancers/cml/), [Chronic myeloid leukaemia, accelerated and blast phase](https://onco.cc/cancers/cml-advanced-phase/), [Chronic myeloid leukaemia, chronic phase](https://onco.cc/cancers/cml-chronic-phase/), [Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL)](https://onco.cc/cancers/all-paediatric-ph-positive/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [ABL1](https://onco.cc/targets/abl1/), [BCR::ABL1 (Philadelphia chromosome)](https://onco.cc/targets/bcr-abl/), [STAT5 (STAT5A, STAT5B)](https://onco.cc/targets/stat5/)
- companies: [Takeda](https://onco.cc/companies/takeda/)
- terms: [BCR::ABL1 kinase domain mutations (T315I and others)](https://onco.cc/terms/abl1-kinase-domain-mutations/), [On-target resistance mutations (gatekeeper, solvent-front, compound)](https://onco.cc/terms/gatekeeper-mutation/), [Ph-positive ALL](https://onco.cc/terms/ph-positive-all/), [Philadelphia chromosome (Ph+, BCR::ABL1)](https://onco.cc/terms/philadelphia-chromosome/)
- trials: [PhALLCON](https://onco.cc/trials/phallcon/), [Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors](https://onco.cc/trials/nct03934372/), [The Study for CML Who Failed Prior TKIs or With T315I Mutation or Ph+ ALL Who Failed Prior TKIs or With T315I Mutation](https://onco.cc/trials/nct04233346/), [Using Tumor Models to Determine Treatments](https://onco.cc/trials/nct06813079/)
- pairings: [BCR::ABL1 TKI + blinatumomab (chemotherapy-free Ph+ ALL)](https://onco.cc/pairings/tki-plus-blinatumomab-ph-all/)
- ideas: [Transplant-free Ph-positive ALL for MRD-negative adults](https://onco.cc/ideas/idea-transplant-free-ph-all/)
- pathways: [BCR::ABL1 (Philadelphia chromosome)](https://onco.cc/pathways/bcr-abl1-signalling/), [Chronic myeloid leukaemia (KEGG map)](https://onco.cc/pathways/cml-signalling/)
- key papers: [PACE: ponatinib in Philadelphia chromosome-positive leukaemias resistant to earlier tyrosine kinase inhibitors](https://onco.cc/key-papers/paper-pace-ponatinib-nejm-2013/)

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