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PONATINIB HYDROCHLORIDE
PONATINIB HYDROCHLORIDE is a drug. It is currently FDA-approved (first approved 2012) for Newly diagnosed Ph+ ALL, Ph+ ALL as monotherapy, CP-CML with resistance or intolerance.
Ponatinib inhibits multiple kinases, including ABL and its T315I mutant, reducing tumor size in mice.
Ponatinib Hydrochloride is a marketed drug primarily indicated for newly diagnosed Ph+ ALL, with a key composition patent expiring in 2028. Its key strength lies in its mechanism of inhibiting multiple kinases, including the ABL T315I mutant, which has shown efficacy in reducing tumor size in preclinical studies. The primary risk is the lack of revenue data and key trial results, which may impact its market positioning and investor confidence.
At a glance
| Generic name | PONATINIB HYDROCHLORIDE |
|---|---|
| Target | ABL, T315I mutant ABL, VEGFR, PDGFR, FGFR, EPH receptors, SRC family, KIT, RET, TIE2, FLT3 |
| Modality | Small molecule |
| Phase | FDA-approved |
| First approval | 2012 |
Mechanism of action
Ponatinib works by blocking the activity of certain enzymes called kinases, which are involved in cell growth and survival. It specifically targets the ABL kinase and its T315I mutant, as well as other kinases like VEGFR, PDGFR, FGFR, and more. By inhibiting these kinases, ponatinib can stop the growth of cancer cells and reduce tumor size.
Approved indications
- Newly diagnosed Ph+ ALL
- Ph+ ALL as monotherapy
- CP-CML with resistance or intolerance
- AP or BP CML with no other options
- T315I-positive CML
Boxed warnings
- WARNING: ARTERIAL OCCLUSIVE EVENTS, VENOUS THROMBOEMBOLIC EVENTS, HEART FAILURE, and HEPATOTOXICITY WARNING: ARTERIAL OCCLUSIVE EVENTS, VENOUS THROMBOEMBOLIC EVENTS, HEART FAILURE, and HEPATOTOXICITY See full prescribing information for complete boxed warning. Arterial occlusive events (AOEs), including fatalities, have occurred in ICLUSIG-treated patients. AOEs included fatal myocardial infarction, stroke, stenosis of large arterial vessels of the brain, severe peripheral vascular disease, and the need for urgent revascularization procedures. Patients with and without cardiovascular risk factors, including patients age 50 years or younger, experienced these events. Monitor for evidence of AOEs. Interrupt or discontinue ICLUSIG based on severity. Consider benefit-risk to guide a decision to restart ICLUSIG. ( 2.2 , 5.1 ) Venous thromboembolic events (VTEs) have occurred in ICLUSIG-treated patients. Monitor for evidence of VTEs. Interrupt or discontinue ICLUSIG based on severity. ( 2.2 , 5.2 ) Heart failure, including fatalities, occurred in ICLUSIG-treated patients. Monitor for heart failure and manage patients as clinically indicated. Interrupt or discontinue ICLUSIG for new or worsening heart failure. ( 2.2 , 5.3 ) Hepatotoxicity, liver failure and death have occurred in ICLUSIG-treated patients. Monitor liver function tests. Interrupt or discontinue ICLUSIG based on severity. ( 2.2 , 5.4 ) Arterial Occlusive Events: Arterial occlusive events (AOEs), including fatalities, have occurred in ICLUSIG-treated patients. AOEs included fatal myocardial infarction, stroke, stenosis of large arterial vessels of the brain, severe peripheral vascular disease, and the need for urgent revascularization procedures. Patients with and without cardiovascular risk factors, including patients age 50 years or younger, experienced these events. Monitor for evidence of AOEs. Interrupt or discontinue ICLUSIG based on severity. Consider benefit-risk to guide a decision to restart ICLUSIG [see Dosage and Administration (2.2) , Warnings and Precautions (5.1) ] . Venous Thromboembolic Events: Venous thromboembolic events (VTEs) have occurred in ICLUSIG-treated patients. Monitor for evidence of VTEs. Interrupt or discontinue ICLUSIG based on severity [see Dosage and Administration (2.2) , Warnings and Precautions (5.2) ] . Heart Failure: Heart failure, including fatalities, occurred in ICLUSIG-treated patients. Monitor for heart failure and manage patients as clinically indicated. Interrupt or discontinue ICLUSIG for new or worsening heart failure [see Dosage and Administration (2.2) , Warnings and Precautions (5.3) ] . Hepatotoxicity: Hepatotoxicity, liver failure and death have occurred in ICLUSIG-treated patients. Monitor liver function tests. Interrupt or discontinue ICLUSIG based on severity [see Dosage and Administration (2.2) , Warnings and Precautions (5.4) ] .
Common side effects
- Rash and related conditions
- Hypertension
- Arthralgia
- Abdominal pain
- Headache
- Constipation
- Dry skin
- Fatigue
- Fluid retention and edema
- Pyrexia
- Nausea
- Pancreatitis/lipase elevation
Drug interactions
- Strong CYP3A Inhibitors
- Strong CYP3A Inducers
Key clinical trials
- Testing the Use of Steroids and Tyrosine Kinase Inhibitors With Blinatumomab or Chemotherapy for Newly Diagnosed BCR-ABL-Positive Acute Lymphoblastic Leukemia in Adults (PHASE3)
- Inotuzumab Ozogamicin and Blinatumomab With or Without Ponatinib in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia (PHASE2)
- Dose-Adjusted EPOCH With or Without Rituximab Plus Ponatinib for the Treatment of Newly-Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia/Lymphoma (PHASE2)
- A Study to Investigate LP-118, Ponatinib, Vincristine and Dexamethasone in Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LBL) (PHASE1, PHASE2)
- Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML) to Characterize the Efficacy and Safety of a Range of Doses (PHASE2)
- 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 (PHASE2)
- Ponatinib Plus Chemotherapy in Acute Lymphoblastic Leukemia Patients (PHASE2)
- A Phase II Study of the Combination of Ponatinib With Mini-hyper CVD Chemotherapy and Venetoclax in Patients With Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PONATINIB HYDROCHLORIDE CI brief — competitive landscape report
- PONATINIB HYDROCHLORIDE updates RSS · CI watch RSS
Frequently asked questions about PONATINIB HYDROCHLORIDE
What is PONATINIB HYDROCHLORIDE?
PONATINIB HYDROCHLORIDE is a Small molecule drug, indicated for Newly diagnosed Ph+ ALL, Ph+ ALL as monotherapy, CP-CML with resistance or intolerance.
How does PONATINIB HYDROCHLORIDE work?
Ponatinib inhibits multiple kinases, including ABL and its T315I mutant, reducing tumor size in mice.
What is PONATINIB HYDROCHLORIDE used for?
PONATINIB HYDROCHLORIDE is indicated for Newly diagnosed Ph+ ALL, Ph+ ALL as monotherapy, CP-CML with resistance or intolerance, AP or BP CML with no other options, T315I-positive CML.
When was PONATINIB HYDROCHLORIDE approved?
PONATINIB HYDROCHLORIDE was first approved on 2012.
What development phase is PONATINIB HYDROCHLORIDE in?
PONATINIB HYDROCHLORIDE is FDA-approved (marketed).
What are the side effects of PONATINIB HYDROCHLORIDE?
Common side effects of PONATINIB HYDROCHLORIDE include Rash and related conditions, Hypertension, Arthralgia, Abdominal pain, Headache, Constipation.
What does PONATINIB HYDROCHLORIDE target?
PONATINIB HYDROCHLORIDE targets ABL, T315I mutant ABL, VEGFR, PDGFR, FGFR, EPH receptors, SRC family, KIT, RET, TIE2, FLT3.
Related
- Target: All drugs targeting ABL, T315I mutant ABL, VEGFR, PDGFR, FGFR, EPH receptors, SRC family, KIT, RET, TIE2, FLT3
- Indication: Drugs for Newly diagnosed Ph+ ALL
- Indication: Drugs for Ph+ ALL as monotherapy
- Indication: Drugs for CP-CML with resistance or intolerance
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing