Ponatinib
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.
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.
1.Ponatinib binds the ATP pocket of BCR::ABL1, including T315I mutants
- Route
- Oral
- Schedule
- 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)
- Monitoring
- Arterial occlusive events, venous thromboembolism, heart failure, hepatotoxicity (boxed warnings); blood pressure; lipase
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
- Medicare
- Part D (self-administered)
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).
- Commercial insurance
- covered with prior authorisation
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. T315I mutation or failure/intolerance of two prior TKIs, per the label.
- Assistance programmes
- Takeda Oncology Here2Assist
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
- Leukemia & Lymphoma Society financial support
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Appraised for
- CML or Ph-positive ALL resistant to dasatinib or nilotinib, or with T315I
- NHS England
- Routinely funded for the appraised indication (or via managed access)
Sources: NICE TA451 · SMC advice: ponatinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
- 14 Dec 2012ApprovalUS
Resistant CML/Ph+ ALL
- 31 Oct 2013WithdrawalUS
Temporary marketing suspension for arterial occlusion; returned with narrowed label December 2013
- 19 Mar 2024ApprovalUS
Frontline Ph+ ALL (PhALLCON), accelerated
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2012 | CML and Ph+ ALL resistant to prior TKIs or with T315I |
| US | 2024 | Newly diagnosed Ph+ ALL with chemotherapy (accelerated) |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Arterial occlusive events CML long-term data at 45 mg; lower with response-based dose reduction | 26% | — |
| Hypertension | 68% | — |
| Pancreatitis / lipase elevation | 40% | — |
| Hepatotoxicity Boxed warning | — | — |
Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
Latest papers
topQuery for this drug: (TITLE:"Ponatinib" OR ABSTRACT:"Ponatinib" OR TITLE:"Iclusig" OR ABSTRACT:"Iclusig") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Ponatinib, not a curated reading list.
Pages like this
not linked directly; found by shared links- ProductDasatinib
Shares BCR::ABL1 TKI + blinatumomab (chemotherapy-free Ph+ ALL), Transplant-free Ph-positive ALL for MRD-negative adults, Ph-positive ALL, Philadelphia chromosome (Ph+, BCR::ABL1).
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Shares On-target resistance mutations (gatekeeper, solvent-front, compound), Ph-positive ALL, Philadelphia chromosome (Ph+, BCR::ABL1), BCR::ABL1 (Philadelphia chromosome).
- TrialD-ALBA (GIMEMA LAL2116)
Shares BCR::ABL1 TKI + blinatumomab (chemotherapy-free Ph+ ALL), Transplant-free Ph-positive ALL for MRD-negative adults, Ph-positive ALL, BCR::ABL1 (Philadelphia chromosome).
- ProductBosutinib
Shares BCR::ABL1 (Philadelphia chromosome), Chronic myeloid leukaemia (CML), Small-molecule kinase inhibitors.
- ProductBlinatumomab
Shares BCR::ABL1 TKI + blinatumomab (chemotherapy-free Ph+ ALL), Transplant-free Ph-positive ALL for MRD-negative adults, Ph-positive ALL, Philadelphia chromosome (Ph+, BCR::ABL1).
- PersonBud Romine
Shares BCR::ABL1 (Philadelphia chromosome), Chronic myeloid leukaemia (CML), Small-molecule kinase inhibitors.
- InstitutionOHSU Knight Cancer Institute
Shares BCR::ABL1 (Philadelphia chromosome), Chronic myeloid leukaemia (CML), Small-molecule kinase inhibitors.
- ProductNilotinib
Shares BCR::ABL1 (Philadelphia chromosome), Chronic myeloid leukaemia (CML), Small-molecule kinase inhibitors.