MidostaurinRydapt
Treatment of adult patients with advanced systemic mastocytosis (AdvSM), including aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), and mast cell leukaemia (MCL).
Decisions on record
- Meeting Mar 2025 Recommended Advanced systemic mastocytosis
- Meeting Jul 2018 Recommended Acute myeloid leukaemia (AML)
- Meeting Nov 2017 Not recommended Acute myeloid leukaemia (AML)
Access path
- TGA registered · Rydapt
TGA label narrower than the PBS population
- Nov 2017Not recommended
vs placebo (chemotherapy alone)
- ↻ resubmittedJul 2018Recommended · restricted
Comparator changed: placebo (chemotherapy alone) → placebo in combination with…
- Mar 2025Recommended · restricted
Comparator changed: placebo in combination with chemotherapy → supportive care (standard…
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Section 100 (Highly Specialised Drugs Program [HSD]) listing of midostaurin for treatment of adult patients with advanced systemic mastocytosis (AdvSM). The PBAC is satisfied that midostaurin provides, for some patients, a significant improvement in efficacy over supportive care (i.e., standard of care [SOC]).PSD · Mar 2025
The PBAC noted the input from the sponsor hearing along with that from the Leukaemia Foundation which highlighted the need for new treatment options in this rare disease that was associated debilitating symptoms and a poor prognosis. The PBAC acknowledged that there was a clear unmet need for treatment options in this rare disease.PSD · Mar 2025
The submission presented a stepped, modelled economic evaluation based on non- randomised studies. The type of economic evaluation presented was a cost-utility analysis. 24PSD · Mar 2025
Table 7: Summary of model structure, key inputs and rationale Component Summary Treatments Midostaurin vs SOC (comprising of cladribine and peginterferon alpha-2a) 20 years in the model base case vs. median follow up of 76 months and median OS of 26.8 months in Time horizon D2201 Outcomes LYG, QALYs gained Methods used to PSM generate results Health states PF response, PF without response, PD, death. Cycle length 28 daysPSD · Mar 2025
standard of care based on ORR and OS, and non-inferior safety.PSD · Mar 2025
The submission described midostaurin as superior in terms of effectiveness compared with SOC. The evaluation considered this claim was not adequately supported. The key issues were:PSD · Mar 2025
The PBAC noted and welcomed the input from the Leukaemia Foundation via the Consumer Comments facility on the PBS website. The comments described the poor prognosis for AdvSM and the need for new treatment options in this rare disease. The comments noted that the only drug on the PBS for systemic mastocytosis (imatinib) was not suitable for the large proportion of AdvSM patients who carry the KIT D816V mutation (see paragraph 5.2).PSD · Mar 2025
No risk-sharing arrangements were proposed in the submission. For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Mar 2025
Cost-effectiveness
ICER value redacted/not published in public summary document
Decision context
PopulationAdult patients (≥18 years) with advanced systemic mastocytosis, including aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL), with no prior treatment with midostaurin for this condition.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | supportive care (standard of care) | — | RCT · ORR |
| Jul 2018 | Recommended · restricted | placebo in combination with chemotherapy | — | RCT · OS |
| Nov 2017 | Not recommended | placebo (chemotherapy alone) | — | RCT · OS |
Consumer voice
The Leukaemia Foundation provided consumer input describing the poor prognosis of advanced systemic mastocytosis, the unsuitability of the only available PBS drug (imatinib) for patients with KIT D816V mutation, and the benefits of midostaurin treatment including reducing end-organ damage, decreasing bone marrow mast cell burden, and improving overall survival.
The comments described the poor prognosis for AdvSM and the need for new treatment options in this rare disease. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line treatment with no prior midostaurin exposure; TGA label has no treatment-history restriction.