Drugs / Ponatinib
last change Jul 2026 re-read 2 minutes ago

Ponatinib

Small molecule targets ABL1, BCR, FLT3, KIT, RET, SRC via inhibition

Developed for
acute lymphoblastic leukemia · chronic myeloid leukemia · B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) · acute myeloid leukemia · blast phase chronic myelogenous leukemia, BCR-ABL1 positive · leukemia, myeloid, accelerated-phase · adult acute lymphoblastic leukemia · non-small cell lung carcinoma
+24 more · Philadelphia-positive myelogenous leukemia · chronic myelomonocytic leukemia · gastrointestinal stromal tumor · lymphoblastic lymphoma · acute leukemia · acute leukemia of ambiguous lineage · B-cell acute lymphoblastic leukemia · B-lymphoblastic leukemia/lymphoma, BCR-ABL1–like · breast cancer · cancer · childhood acute lymphoblastic leukemia · digestive system cancer · endometrium neoplasm · glioblastoma · hepatobiliary neoplasm · leukemia · lung adenocarcinoma · medullary thyroid gland carcinoma · mixed phenotype acute leukemia with t(9;22)(q34.1;q11.2) · pancreatic ductal adenocarcinoma · precursor lymphoblastic lymphoma/leukemia · small cell lung carcinoma · T-cell acute lymphoblastic leukemia · thyroid cancer
Investigated by
Ariad Pharmaceuticals · M.D. Anderson Cancer Center · National Cancer Institute (NCI) · Takeda · GIMEMA Foundation · OHSU Knight Cancer Institute
+53 more · Versailles Hospital · Amgen · Dana-Farber Cancer Institute · Incyte Corporation · Nanfang Hospital, Southern Medical University · Oregon Health and Science University · Peking University People's Hospital · Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University · Third Affiliated Hospital, Sun Yat-Sen University · University of Chicago · University of Washington · Antonio Fojo · Apices Soluciones S.L. · Assistance Publique - Hôpitaux de Paris · Associazione Italiana Pazienti Leucemia Mieloide Cronica · CRIS Cancer Foundation · CZECRIN - Czech Clinical Research Infrastructure Network · Cardiff University · Centre Leon Berard · Deutsche Leukämie- & Lymphom-Hilfe · Fondazione per la Medicina Personalizzata · Fundación Teófilo Hernando, Spain · GWT-TUD GmbH · German Federal Ministry of Education and Research · Goethe University · Guangdong Provincial People's Hospital · Guangxi Medical University · Guangzhou First People's Hospital · Guangzhou General Hospital of Guangzhou Military Command · Hannover Medical School · Helios Klinikum Bad Saarow · Helios Klinikum Berlin-Buch · Incyte Biosciences UK · Incyte Corporation · Institute of Hematology and Blood Transfusion, Czech Republic · Massachusetts General Hospital · Mayo Clinic · Memorial Sloan Kettering Cancer Center · Millennium Pharmaceuticals, Inc. · Otsuka Beijing Research Institute · PETHEMA Foundation · Sameek Roychowdhury · Sebastian Bauer · University Health Network, Toronto · University Hospital Tuebingen · University Hospital, Aachen · University Medical Center Mannheim · University of Alberta · University of Colorado, Denver · University of Michigan Rogel Cancer Center · Washington University School of Medicine · WiSP GmbH · Zhujiang Hospital

Regulatory milestones approvals, filings & regulatory actions · 8 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (6) US (FDA) ICLUSIG 2016-06-02 – 2025-10-10 fda.gov
Approved EU (EMA) Iclusig 2013-07-01 europa.eu
Approved US (FDA) ICLUSIG 2012-12-14 fda.gov

Trials 65 · started per year

today
US approved 2012
EU approved 2013
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200820092010201120122013201420152016201720182019202020212022202320242025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 13 trials
Phase 1 NCT03878524 Apr → Dec 2020 acute myeloid leukemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, blast phase chronic myelogenous leukemia, BCR-ABL1 positive, castration-resistant prostate carcinoma +14 OHSU Knight Cancer Institute Terminated No outcome recorded
Phase 1 NCT02779283 Jan 2016 → Apr 2018 acute lymphoblastic leukemia, acute myeloid leukemia OHSU Knight Cancer Institute Completed No outcome recorded
Phase 1 NCT00660920 Jun 2008 → May 2016 chronic myeloid leukemia Ariad Pharmaceuticals Completed No outcome recorded
Phase 1/28 trials
Phase 1/2 NCT06207123 Sep 2024 → Dec 2026 expected acute leukemia, lymphoblastic lymphoma University of Chicago Recruiting No outcome recorded
Phase 1/2 NCT04501614 Feb 2021 → Dec 2023 B-lymphoblastic leukemia/lymphoma, BCR-ABL1–like, childhood acute lymphoblastic leukemia, mixed phenotype acute leukemia with t(9;22)(q34.1;q11.2) Takeda Terminated No outcome recorded
Phase 1/2 NCT03934372 Jan 2020 → Feb 2028 expected acute lymphoblastic leukemia, acute myeloid leukemia, blast phase chronic myelogenous leukemia, BCR-ABL1 positive, leukemia, myeloid, accelerated-phase Incyte Corporation Recruiting No outcome recorded
Phase 1/2 NCT03704688 Oct 2018 → Feb 2022 non-small cell lung carcinoma Memorial Sloan Kettering Cancer Center Completed No outcome recorded
Phase 1/2 NCT03576547 Jun 2018 → Jun 2024 Philadelphia-positive myelogenous leukemia, acute lymphoblastic leukemia, blast phase chronic myelogenous leukemia, BCR-ABL1 positive M.D. Anderson Cancer Center Terminated No outcome recorded
Phase 1/2 NCT02829840 Sep 2016 → Sep 2021 acute myeloid leukemia M.D. Anderson Cancer Center Withdrawn No outcome recorded
Phase 1/2 NCT02428543 Jul 2013 → Oct 2021 acute myeloid leukemia Versailles Hospital Completed No outcome recorded
Phase 1/2 NCT01667133 Aug 2012 → Aug 2018 acute lymphoblastic leukemia, chronic myeloid leukemia Ariad Pharmaceuticals Completed No outcome recorded
Phase 242 trials
Phase 2 NCT07301424 Jul 2026 → Dec 2027 expected B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) University of Alberta Not yet recruiting No outcome recorded
Phase 2 NCT07224100 Mar 2026 → Jul 2028 expected B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2), lymphoblastic lymphoma University of Washington Recruiting No outcome recorded
Phase 2 NCT06813079 Apr 2025 → Feb 2028 expected pancreatic ductal adenocarcinoma University Health Network, Toronto Recruiting No outcome recorded
Phase 2 NCT04845035 Jan 2024 → Jan 2026 acute lymphoblastic leukemia University of Michigan Rogel Cancer Center Withdrawn No outcome recorded
Phase 2 NCT06061094 Jul 2023 → Jul 2029 expected B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) Goethe University Recruiting No outcome recorded
Phase 2 NCT05306301 Oct 2022 → Mar 2026 overdue acute lymphoblastic leukemia GIMEMA Foundation Active not recruiting No outcome recorded
Phase 2 NCT05268003 Jun 2022 → Apr 2026 T-cell acute lymphoblastic leukemia M.D. Anderson Cancer Center Terminated No outcome recorded
Phase 2 NCT04475731 May 2021 → Nov 2024 overdue B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2), acute lymphoblastic leukemia GIMEMA Foundation Active not recruiting No outcome recorded
Phase 2 NCT04747912 Mar 2021 → Mar 2028 expected B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2), acute lymphoblastic leukemia University of Chicago Recruiting No outcome recorded
Phase 2 NCT04554459 Feb 2021 → Sep 2023 B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2), adult acute lymphoblastic leukemia Institute of Hematology and Blood Transfusion, Czech Republic Terminated No outcome recorded
Phase 2/33 trials
Phase 2/3 NCT06860269 May 2025 → Mar 2035 expected acute lymphoblastic leukemia Assistance Publique - Hôpitaux de Paris Recruiting No outcome recorded
Phase 2/3 NCT03624530 Aug 2018 → Jul 2021 B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) Nanfang Hospital, Southern Medical University Unknown No outcome recorded
Phase 2/3 NCT01761747 Jan 2013 → Jun 2014 non-small cell lung carcinoma Dana-Farber Cancer Institute Terminated No outcome recorded
Phase 35 trials
Phase 3 NCT04722848 Sep 2021 → Sep 2027 expected adult acute lymphoblastic leukemia GIMEMA Foundation Active not recruiting No outcome recorded
Phase 3 NCT04530565 Jan 2021 → Jul 2028 expected B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2) National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 3 NCT03589326 Oct 2018 → Aug 2022 overdue acute lymphoblastic leukemia Takeda Active not recruiting No outcome recorded
Phase 3 NCT02627677 Dec 2015 → Oct 2020 chronic myeloid leukemia Ariad Pharmaceuticals Terminated No outcome recorded
Phase 3 NCT01650805 Jun 2012 → Oct 2013 chronic myeloid leukemia Ariad Pharmaceuticals Terminated No outcome recorded
Phase not applicable2 trials
NCT01888562 Jan 2016 → Jul 2018 endometrium neoplasm Washington University School of Medicine Withdrawn No outcome recorded
NCT02551718 Sep 2015 → Jun 2020 acute leukemia of ambiguous lineage, acute lymphoblastic leukemia University of Washington Completed No outcome recorded
Phase not stated2 trials
NCT01549548 Philadelphia-positive myelogenous leukemia, chronic myeloid leukemia OHSU Knight Cancer Institute No longer available No outcome recorded
NCT01592136 acute lymphoblastic leukemia, chronic myeloid leukemia Ariad Pharmaceuticals Approved for marketing No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 2 NCT01838642 Comparator Mar 2013 → Dec 2015 thyroid cancer National Institutes of Health Clinical Center (CC) Terminated No outcome recorded

Press releases naming this drug 14 releases

DateIssuerRelease
2026-03-27 Otsuka Pharmaceutical Development & Commercialization, Inc. Otsuka Submits Application in Japan for Iclusig for First-Line Treatment of Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia® otsuka.co.jp
2024-03-19 Takeda Takeda Announces U.S. FDA Approval of Supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) in Adult Patients with Newly Diagnosed Ph+ ALL takeda.com
2022-11-17 Takeda Phase 3 Trial of ICLUSIG ® (ponatinib) Met Primary Endpoint in Newly-Diagnosed Ph+ ALL, a Setting with No Targeted Treatments Approved in the US takeda.com
2021-05-20 Incyte Corporation Positive Primary Analysis from the Phase 2 OPTIC Study of Ponatinib (Iclusig®) in Chronic Phase-Chronic Myeloid Leukemia (CP-CML) to be Presented at the 2021 ASCO Annual Meeting incyte.com
2021-05-19 Takeda Takeda to Present Positive Primary Analysis from Phase 2 OPTIC Trial of ICLUSIG® (ponatinib), Reinforcing Ability to Address Gaps in Care for Chronic-Phase CML takeda.com
2020-12-18 Takeda U.S. FDA Approves Supplemental New Drug Application for Takeda’s ICLUSIG ® (ponatinib) for Adult Patients with Resistant or Intolerant Chronic-Phase CML takeda.com
2020-05-29 Takeda Takeda to Present Data from the ICLUSIG ® (ponatinib) Clinical Trial Program that Could Prove Practice-Changing for the Treatment of Chronic-Phase CML takeda.com
2020-05-29 Incyte Corporation Interim Data from the Open Label Phase 2 OPTIC Study of Iclusig® (ponatinib) for Chronic Phase-CML Presented at the ASCO and EHA Virtual Meetings incyte.com
2018-03-22 Takeda Takeda Announces Publication of Final Data from ICLUSIG ® (ponatinib) Pivotal Phase 2 PACE Trial in Blood takeda.com
2016-11-18 Otsuka Pharmaceutical Development & Commercialization, Inc. ICLUSIG Tablets 15 mg Now Available in Japan for Patients with Chronic Myeloid Leukemia and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia® otsuka.co.jp
2016-09-28 Otsuka Pharmaceutical Development & Commercialization, Inc. ICLUSIG Tablets 15 mg Approved in Japan for Patients with Chronic Myeloid Leukemia and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia® otsuka.co.jp
2016-05-09 Incyte Corporation Incyte and ARIAD Announce Agreement for Incyte to Acquire ARIAD’s European Operations and In-license Iclusig® (ponatinib) in Europe incyte.com

Evidence & citations 10 cited values

Every value below carries the sentence it was read from. 69 sources stand behind the page.

FieldValueCited text
Known as Ponatinib “Imatinib was initiated at a dose of 200mg/d, dasatinib at a dose of 50mg/d, and ponatinib at a dose of 30mg/d.” NCT03624530
64

Ponatinib is a drug, taken by mouth, that targets and kills leukemia cells that have the BCR-ABL mutation.” NCT07301424

“If the patients had T315I mutations, ponatinib will be given.” NCT01883219

“Pentostatin Ponatinib Pralatrexate” NCT02551718

NCT01570868

NCT01874665

NCT03171389

NCT04475731

NCT03690115

NCT01667133

NCT01813734

NCT04591431

NCT02779283

NCT04160546

NCT01838642

NCT04188405

NCT06207123

NCT02829840

NCT02467270

NCT00660920

NCT03838692

NCT04070443

NCT04722848

NCT02776605

NCT01761747

NCT01746836

NCT03807479

NCT03895671

NCT01641107

NCT03934372

NCT03704688

NCT03147612

NCT01935336

NCT02428543

NCT03263572

NCT02398825

NCT02265341

NCT03878524

NCT04554459

NCT03739814

NCT06061094

NCT02272998

NCT04709731

NCT05306301

NCT04530565

NCT01592136

NCT03589326

NCT05268003

NCT02627677

NCT01888562

NCT07224100

NCT04688983

NCT06860269

NCT04747912

NCT04233346

NCT04043676

NCT01549548

NCT01424982

NCT04501614

NCT06813079

NCT01620216

NCT04845035

NCT01207440

NCT02478164

NCT01650805

Known as AP-24534 HCL ChEMBL registry synonym — accepted as the source's own label CHEMBL1171837
Known as AP24534 “Ponatinib (AP24534) is a potent oral tyrosine kinase inhibitor that blocks native and mutated BCR-ABL, including the gatekeeper mutant T315I, which is uniformly resistant to...” PMID 23190221 Nov 2012
2

“Ponatinib (AP24534) is a third generation tyrosine kinase inhibitor targeting the BCR-ABL tyrosine kinase domain.” NCT02428543

“Expanded Access Program of Ponatinib (AP24534)” NCT01592136

Known as ICLUSIG “In the Phpos ALL subset, the third-generation tyrosine kinase inhibitor ponatinib (PONA, Iclusig®, Incyte) has also been evaluated frontline with promising results when...” NCT06860269
2

“Ponatinib (Iclusig®) : Tyrosine Kinase Inhibitor (BCR-ABL); oral (tablets) : 30mg/day during 6 months (induction phase)” NCT04070443

“patients will receive a dosage of 15 mg of ICLUSIG (ponatinib).” NCT05306301

Known as PONA “In the Phpos ALL subset, the third-generation tyrosine kinase inhibitor ponatinib (PONA, Iclusig®, Incyte) has also been evaluated frontline with promising results when...” NCT06860269
Known as Ponatinib Hydrochloride 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” NCT01746836
2

“Drug: Ponatinib HydrochlorideNCT02265341

NCT03576547

Action Inhibit “Ponatinib (AP24534) is a potent oral tyrosine kinase inhibitor that blocks native and mutated BCR-ABL, including the gatekeeper mutant T315I, which is uniformly resistant to...” PMID 23190221 Nov 2012
Modality Small molecule “Ponatinib is a novel synthetic orally active tyrosine kinase inhibitor (TKI)” NCT01641107
Route Oral “administered ponatinib 45 mg, tablet, orally” NCT01207440
Target ABL1 “The BCR-ABL1 inhibitor ponatinib is approved for the treatment of adults with chronic myeloid leukemia or Philadelphia chromosome-positive acute lymphoblastic leukemia,...” PMID 34699069 Dec 2021