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Metabolic

Taliglucerase alfa

Brand: Elelyso®

PBAC's latest decision on Taliglucerase alfa: Not recommended (2014). Considered for Long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease.

PBAC outcome
Not recommended
Restricted
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
2
first 2012
Submissions
2
2012 → 2014

Eligible population

Adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopaenia.

Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
RCT
Primary endpoint
Other
Pivotal trial size
31 patients
Key trials
PB-06-001, PB-06-005, PB-06-002, PB-06-003, PB-06-004, PB-06-006, HGT-039, TKT-032, TKT-034, HGT-044
Comparator
imiglucerase and velaglucerase alfa
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis was not accepted because equivalent comparative efficacy and safety were not established.

Why PBAC said no

Reasons cited in the latest PSD: equivalent comparative efficacy and safety to imiglucerase and velaglucerase alfa not established, lack of head-to-head direct randomised trial control data, differences in baseline values and outcome measurements limited comparability between trials, uncertain non-inferiority

Submission history

Similar precedents

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