Daunorubicin With CytarabineVYXEOS
Treatment of newly diagnosed therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC) in adults.
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- Meeting Mar 2024 Withdrawn Acute myeloid leukaemia no PSD
- Meeting Mar 2024 Recommended Acute myeloid leukaemia no PSD
- Meeting Nov 2023 Recommended Therapy-related acute myeloid leukaemia or acute myeloid leukaemia with myelodysplasia-related changes
- Meeting Jul 2023 Not recommended Therapy-related acute myeloid leukaemia (t-AML) and acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC)
- Meeting Jul 2022 Withdrawn Therapy-related acute myeloid leukaemia and acute myeloid leukaemia with myelodysplasia-related changes no PSD
Access path
- TGA registered · VYXEOS
TGA label narrower than the PBS population
- Nov 2023Recommended
vs idarubicin plus cytarabine
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended liposomal daunorubicin and cytarabine for the treatment of therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia-related changes (AML-MRC). The PBAC’s recommendation for listing was based on, among other matters, its assessment that the cost-effectiveness of daunorubicin and cytarabine would be acceptable at the price proposed in the resubmission.PSD · Nov 2023
5.2 The PBAC acknowledged the advice received via the consumer comments facility which noted the unmet need for alternate AML treatments and was supportive of the submission. 5.3 The PBAC recalled that in July 2023 it had:PSD · Nov 2023
4.4 As an early re-entry resubmission, the economic analysis has not been independently evaluated.PSD · Nov 2023
4.5 In July 2023, the PBAC advised that a revised economic model should:PSD · Nov 2023
Source: Table 1-1, p4 of the July 2023 submission For more detail on PBAC’s view, see section 5 PBAC outcome.PSD · Nov 2023
• accepted that daunorubicin was an acceptable proxy for idarubicin when given at the recommended dose for the 7+3 regimen in the induction setting; 5PSD · Nov 2023
4.2 The PBAC noted and welcomed the input from one health care professional (in addition to the 13 health care professionals and 2 organisations who provided advice in July 2023) via the Consumer Comments facility on the PBS website. The comment described the benefits of treatment with liposomal daunorubicin with cytarabine, including improved progression free and overall survival, in patients who received induction and consolidation.PSD · Nov 2023
Cost-effectiveness
Decision context
PopulationAdults with newly diagnosed therapy-related acute myeloid leukaemia or acute myeloid leukaemia with myelodysplasia-related changes, without prior chemotherapy for induction, excluding FLT3 mutation positive patients and those with favourable risk cytogenetics.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2023 | Recommended | idarubicin plus cytarabine | $55k–75k | RCT · OS |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| Study 301 | Ph 3 | 309 | Overall Survival | completed |
Consumer voice
One health care professional provided input via the PBS website Consumer Comments facility, describing the benefits of liposomal daunorubicin with cytarabine treatment, including improved progression-free and overall survival in patients who received induction and consolidation therapy.
The comment described the benefits of treatment with liposomal daunorubicin with cytarabine, including improved progression free and overall survival, in patients who received induction and consolidation. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line, treatment-naive patients, excluding FLT3-positive and favourable-risk cytogenetics cases.