AsciminibScemblix
Addition of nurse practitioners as eligible prescribers for continuing treatment phases of asciminib in patients with Ph+ CML (chronic phase or accelerated phase) who had been previously treated with two or more TKIs, or with the T315I mutation.
Decisions on record
The committee reached more than one outcome for this medicine at its most recent sitting — different indications were decided differently. The header reflects the least favourable of them; all are listed below.
- Meeting Mar 2026 Withdrawn Chronic myeloid leukaemia (CML)
- Meeting Mar 2026 Recommended Chronic myeloid leukaemia (CML)
- Meeting Nov 2022 Recommended Chronic myeloid leukaemia in chronic phase previously treated with tyrosine kinase inhibitors
- Meeting Jul 2022 Not recommended Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase previously treated with two or more tyrosine kinase inhibitors
Access path
- TGA registered · Scemblix
TGA label broader than the PBS population
- Jul 2022Not recommended
vs Nilotinib (primary comparator); ponatinib (supplementary…
- ↻ resubmittedNov 2022Recommended · restricted
Comparator changed: Nilotinib (primary comparator); ponatinib (supplementary comparator)…
- Mar 2026Recommended · restricted
Evidence: RCT → Other
- PBS listing · Restricted
From the public summary
6.1 The PBAC recommended the Authority Required listing of asciminib for the treatment of (i) patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP) or accelerated phase (AP), who had been previously treated with two or more tyrosine kinase inhibitors (TKIs); and (ii) patients with Ph+ CML in CP or AP, who had been previously treated with one or more TKIs and harbouring the T315I mutation.PSD · Nov 2022
6.2 The PBAC considered that the resubmission had addressed the outstanding issues identified at the July 2022 PBAC meeting. The resubmission proposed a separate restriction for patients with the T315I mutation; provided clinical data to support non- inferiority of asciminib to ponatinib in patients with the T315I mutation; provided an economic evaluation reflecting benchmarking against the second generation TKIs nilotinib and dasatinib in patients …PSD · Nov 2022
5.31 As an early re-entry resubmission, the economic analysis has not been independently evaluated.PSD · Nov 2022
5.32 The July 2022 submission presented separate analyses of asciminib versus nilotinib and asciminib versus ponatinib using the cost minimisation approach (CMA). Although the PBAC considered that benchmarking against second generation TKIs was a reasonable basis for establishing a cost-effective price for asciminib in patients without the T315I mutation, it requested that this benchmarking be performed against nilotinib and dasatinib (paragraphs 7.13 and …PSD · Nov 2022
5.28 The clinical claim for asciminib (200 mg twice daily) versus ponatinib (45 mg once daily) in patients with CML-CP who have received at least one prior TKI and harbour the T315I mutation was:PSD · Nov 2022
• Non-inferior efficacy. The assessment of efficacy was based on a comparison of MMR and CCyR in the T315I mutation populations of the X2101 and PACE trials. The Sponsor stated that these outcomes are important treatment goals (NCCN, 2022) and have previously been accepted by the PBAC in their consideration in the treatment of CML (Nilotinib PSD July 2011; Ponatinib PSD July 2015).PSD · Nov 2022
The comments from a HCP described a range of benefits of treatment with asciminb, including effectiveness where other TKIs have failed and a lower level of toxicity than all the currently available TKIs. The HCP considered asciminib to be an essential medication for a small number of patients with CML who have no other options.PSD · Jul 2022
The PBAC noted that The Leukaemia Foundation received feedback from Australian clinicians using asciminib via the ASCEND-CML trial (in combination with imatinib) and the sponsor’s Compassionate Access Scheme, as well as from a patient being treated with the second generation TKIs nilotinib and dasatinib.PSD · Jul 2022
Cost-effectiveness
No economic evaluation submitted; this is a prescriber-type amendment with no anticipated budget impact.
The PBAC considered that there would be no additional cost to the PBS. PBAC · 2026
Decision context
PopulationNurse practitioners providing continuing treatment for patients with Ph+ CML in chronic phase or accelerated phase who had been previously treated with two or more TKIs or with the T315I mutation, where patient care is shared with a medical practitioner (haematologist).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | — | — | Other |
| Nov 2022 | Recommended · restricted | Nilotinib (main comparator for non-T315I patients); Ponatinib (supplementary comparator for T315I patients) | — | RCT · MMR, CCyR |
| Jul 2022 | Not recommended | Nilotinib (primary comparator); ponatinib (supplementary comparator) | — | RCT · MMR |
Consumer voice
No consumer comments were received for this item. The PBAC previously noted input from a health care professional and two organisations (The Leukemia Foundation and Rare Cancers Australia) in relation to asciminib at the July 2022 PBAC meeting.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label broader than PBS population — PBAC includes T315I patients with only one prior TKI; TGA requires two or more TKIs for non-T315I patients.