PBAC's latest decision on Caplacizumab: Recommended with restriction (2020). Considered for Treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in adults, in conjunction with plasma exchange and immunosuppression.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2020
Submissions
1
2020 → 2020
Eligible population
Adults (18 years or older) with acquired thrombotic thrombocytopenic purpura (aTTP) with ADAMTS13 activity <10%.
Therapy area
Haematology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Time to platelet normalisation, TTP-related death, TTP recurrence, TTP relapse, major thromboembolic events, refractory TTP, time to normalisation of organ damage markers
Key trials
HERCULES, TITAN
Comparator
plasma exchange and immunosuppression (standard of care)
Economic model
CUA
ICER note
ICER not explicitly stated in the public document; pricing information is redacted.