Drug · Small Molecule

Ponatinib

CI-DRUG-00000483Explore in graph →NCIt C95777 CHEMBL1171837 CIViC Approved

Regulatory

Approvals (10)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

health-canada-dpdRegulatory10 approval records from 3 sources (hover a row badge for its dataset) · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
CA Health Canada	—	Marketed in Canada as ICLUSIG (DIN 02437341) under ATC L01EA05 PONATINIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	withdrawnsource: Dormantsince Aug 8, 2022	Jan 7, 2016	health-canada-dpd
CA Health Canada	—	Marketed in Canada as ICLUSIG (DIN 02437333) under ATC L01EA05 PONATINIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Aug 21, 2015	health-canada-dpd
EU EMA	—	Iclusig is indicated as monotherapy in adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation. Iclusig is indicated in combination with reduced-intensity chemotherapy in adult patients with newly diagnosed Ph+ ALL (see section 5.1).  Iclusig is indicated as monotherapy in paediatric patients 6 years of age or older with chronic phase chronic myeloid leukaemia (CP-CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation. See sections 4.2 for the assessment of cardiovascular status prior to start of therapy and 4.4 for situations where an alternative treatment may be considered.

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 2 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 3	17	9	5	55	10	CA, EU, US	Dec 14, 2012	2008-06
Leukemia	Phase3	Phase 3	12	6	2	36	0	—	—	2008-06
Malignant Lung Neoplasm	Phase3	Phase 3	0	0	1	5	0	—	—	2013-01
Non-Hodgkin Lymphoma	Phase2	Phase 2	2	2	0	3	0	—	—

Curated evidence

Clinical evidence (69)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–50 of 69 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
ABL1 T315I + ABL1 Fusion7
Axitinib + Ponatinib

Clinical trials

Trials with this intervention (64)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–50 of 64 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT03739814	Inotuzumab Ozogamicin and Blinatumomab With or Without Ponatinib in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia	recruiting	Phase 2	84	National Cancer Institute (NCI)NIH	2	Sep 24, 2026	clinicaltrials
NCT01746836	Ponatinib Hydrochloride as Second Line Therapy in Treating Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib Mesylate, Dasatinib, or Nilotinib

Molecule

Record

Kind: Small Molecule
Mechanism: Bcr/Abl fusion protein inhibitor
Targets: 2 genes
UNII: 96R6PU3D8J
Aliases: AP 24534, AP-24534, AP24534, Benzamide, 3-(2-Imidazo(1,2-B)Pyridazin-3-Ylethynyl)-4-Methyl-N-(4-((4-Methyl-1- Piperazinyl)Methyl)-3-(Trifluoromethyl)Phenyl), ICLUSIG, Ponatinib, Ponatinib Hydrochloride

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (6)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01EA05	PONATINIB
DIN (Health Canada)	02437333	ICLUSIG
DIN (Health Canada)

FLT3 ITD2
AG1295 + AGS324 + Crenolanib + FLT3 Tyrosine Kinase Inhibitor TTT-3002 + KW2449 + Lestaurtinib + Linifanib + Midostaurin + Ponatinib + Quizartinib + R406 + Sorafenib + SunitinibSubstitutes	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	submitted	EID11084 A panel of FLT3 tyrosine kinase inhibitors was tested against Ba/F3 cells transfected with FLT3-ITD using MTT growth/survival assay. Each of the 13 TKIs inhibited proliferation of FLT3-ITD cells in a … (full text at CIViC) PMID 28077790 · Nguyen et al., 2017 · Open in CIViC	civic
Ponatinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	5	accepted	EID7369 In a preclinical trial, multiple experiments testing the effectiveness of ponatinib on the mutation FLT3-ITD in acute myeloid leukemia (AML) were conducted. Ponatinib was very effective in targeting t… (full text at CIViC) PMID 21482694 · Gozgit et al., 2011 · Open in CIViC	civic

FLT3 Y572del2
Crenolanib + Lestaurtinib + Midostaurin + Ponatinib + Quizartinib + SorafenibSubstitutes	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	5	submitted	EID9306 The construct supported IL-3 independent growth of Ba/F3 cells. It showed FLT-3 autophosphorylation and downstream signaling via STAT5, AKT, and ERK1/2 pathways. With IL-3 deprivation, the cells showe… (full text at CIViC) PMID 33914060 · Young et al., 2021 · Open in CIViC	civic
Ponatinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	submitted	EID12864 Ba/F3 cell lines expressing the variant FLT3 Y572del showed sensitivity to ponatinib comparable to the FLT3-ITD construct (Figure 4). PMID 33914060 · Young et al., 2021 · Open in CIViC	civic

KIT N822K3
Ponatinib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	2	accepted	EID7379 In this preclinial trial, the AML cell line Kasumi-1 with c-KIT N822K mutation was treated with Ponatinib. This drug effectively inhibits both KIT phosphorylation and viability for Kasumi-1 cells at I… (full text at CIViC) PMID 21482694 · Gozgit et al., 2011 · Open in CIViC	civic
〃	Gastrointestinal Stromal Tumor	Predictive	D	Does Not Support Resistance	3	accepted	EID4167 A preclinical study tested the effect of a tyrosine kinase inhibitor (TKI) on Ba/F3 cell lines with primary ponatinib-sensitizing del 557-558 mutation, which induced IL-3 independent growth, and secon… (full text at CIViC) PMID 25239608 · Garner et al., 2014 · Open in CIViC	civic
〃	Gastrointestinal Stromal Tumor	Predictive	D	Supports Sensitivity Response	4	submitted	EID7408 Ponatinib possesses potent activity against N882K KIT mutant in preclinical experiments. PMID 25239608 · Garner et al., 2014 · Open in CIViC	civic

ABL1 Fusion2
Ponatinib	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	3	accepted	EID9213 Authors reported a 26 year-old female patient with B-ALL and RCSD1-ABL1 fusion identified by FISH and PCR. Sequencing showed that RCSD1 exon 3 was fused to ABL1 exon 4. This patient was treated with c… (full text at CIViC) PMID 25768406 · Collette et al., 2015 · Open in CIViC	civic
〃	B Lymphoblastic Leukemia/LymphomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	3	rejected	EID9214 The authors performed an ex vivo functional evaluation of the drug response profiling (DRP) of the leukemic blasts from a B-ALL patient with RCSD1-ABL1 fusion with a specific focus on TKIs. Resistance… (full text at CIViC) PMID 25768406 · Collette et al., 2015 · Open in CIViC	civic

KIT A829P2
Ponatinib	Gastrointestinal Stromal Tumor	Predictive	D	Supports Sensitivity Response	3	submitted	EID5862 Ponatinib possesses potent activity against most major clinically-relevant KIT mutants, and has demonstrated preliminary evidence of activity in patients with refractory GIST. These data strongly supp… (full text at CIViC) PMID 25239608 · Garner et al., 2014 · Open in CIViC	civic
〃	Gastrointestinal Stromal Tumor	Predictive	D	Does Not Support Resistance	4	accepted	EID7413 Ponatinib possesses potent activity against A829P KIT mutant cells in preclinical experiments. A preclinical study tested the effect of a tyrosine kinase inhibitor (TKI) on Ba/F3 cell lines with repre… (full text at CIViC) PMID 25239608 · Garner et al., 2014 · Open in CIViC	civic