PonatinibIclusig
Chronic myeloid leukaemia (CML) in patients with T315I mutation who have failed prior tyrosine kinase inhibitor therapy, or in patients where both nilotinib and dasatinib have failed or where one has failed and the patient is intolerant of the other.
Decisions on record
- Meeting Nov 2018 Recommended Chronic myeloid leukaemia (CML)
- Meeting Apr 2018 Recommended Acute lymphoblastic leukaemia no PSD
- Meeting Nov 2017 Recommended Relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukaemia (ALL)
- Meeting Jul 2015 Recommended Chronic myeloid leukaemia (CML) and acute lymphoblastic leukaemia (ALL)
- Meeting Nov 2014 Deferred 15 mg tablet, 60 45 mg tablet, 30 Iclusig® Specialised Therapeutics Australia Pty Ltd New Listing (Major submission) Leukaemia Authority Required listing for ponatinib for treatment of adult patients: - with chronic myeloid leukaemia (CML) who are resistant or intolerant to dasatinib or nilotinib, o
Access path
- TGA registered · Iclusig
TGA label equal than the PBS population
- Nov 2014Deferred
vs dasatinib and nilotinib; best supportive care and…
- Jul 2015Recommended · restricted
Comparator changed: dasatinib and nilotinib; best supportive care and allogeneic stem…
- Nov 2017Recommended
Comparator changed: dasatinib and nilotinib → dasatinib
- Nov 2018Recommended
Evidence: Registry → Other
- PBS listing · Authority Required
From the public summary
6.1 The PBAC recommended an amendment to the existing PBS initial treatment restrictions for ponatinib for chronic myeloid leukaemia (CML), to remove reference to the six month timeframe between the bone marrow biopsy pathology report(s) and the application for initiation of treatment. The PBAC noted this amendment aligns with the current requirements for other tyrosine kinase inhibitors used in second- and third-line treatment of CML.PSD · Nov 2018
6.2 The PBAC also recommended an amendment to the existing PBS continuing treatment restriction for ponatinib for CML, to enable telephone authority for subsequent continuing treatment. The PBAC noted that a new restriction for the subsequent continuing treatment phase would need to be added to the existing prescribing rule to implement this change.PSD · Nov 2018
6.19 The re-submission presented a cost-minimisation analysis for ponatinib. This was similar to the previous submission. The re-submission updated the equi-effective dose compared with the previous submission to reflect the PBAC recommendation. 6.20 The equi-effective doses were estimated as:PSD · Jul 2015
Ponatinib 30.2 mg daily = Dasatinib 102 mg daily = Nilotinib 797 mg daily These were consistent with the previous PBAC consideration.PSD · Jul 2015
6.17 The re-submission described ponatinib as non-inferior in terms of comparative effectiveness and inferior in terms of comparative safety over dasatinib and nilotinib.PSD · Jul 2015
In the previous submission, the re-submission made the same claim in terms of comparative effectiveness and no claim in terms of comparative safety. The clinical claim in the re-submission was consistent with the PBAC recommendation.PSD · Jul 2015
4.3 The current minor submission did not present new data for the use of ponatinib in the treatment of refractory Ph+ALL, but considered that further scrutiny of existing data demonstrated that the treatment benefit in patients with and without the T315I mutation is comparable.PSD · Nov 2017
Cost-effectiveness
Minor submission with no economic evaluation required; changes to existing PBS restrictions only.
The current minor submission did not present new data for the use of ponatinib in the treatment of refractory Ph+ALL, but considered that further scrutiny of existing data demonstrated that the treatment benefit in patients with and without the T315I mutation is comparable. PSD · 2017
Decision context
PopulationPatients with chronic myeloid leukaemia with T315I mutation who have failed prior imatinib, dasatinib, or nilotinib therapy, or patients where both nilotinib and dasatinib have failed or where one has failed and the patient is intolerant of the other.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2018 | Recommended | — | — | Other |
| Nov 2017 | Recommended | dasatinib | — | Registry |
| Jul 2015 | Recommended · restricted | dasatinib and nilotinib | — | Single-arm · ORR |
| Nov 2014 | Deferred | dasatinib and nilotinib; best supportive care and allogeneic stem cell transplantation in select populations | — | Single-arm · ORR |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| PACE | Ph 2 | 449 | Percentage of CP-CML Participants With Major Cytogenetic Response (MCyR) | completed |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both describe Ph+ ALL patients failing/intolerant to dasatinib without T315I mutation; no meaningful restriction differentiates them.