PBAC's latest decision on Midostaurin: Recommended with restriction (2025). Considered for 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).
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
3
first 2017
Submissions
3
2017 → 2025
Eligible population
Adult 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.
Therapy area
Haematology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
ORR
Key trials
D2201, A2213
Comparator
supportive care (standard of care)
Economic model
CUA
ICER note
ICER value redacted/not published in public summary document
Submission history
Nov 2017: Recommended with restriction · Authority Required
Jul 2018: Recommended with restriction · Restricted
Mar 2025: Recommended with restriction · Authority Required