PBAC's latest decision on Vutrisiran: Recommended with restriction (2024). Considered for Treatment of hereditary transthyretin mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, defined by Familial Amyloid Neuropathy (FAP) stage.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
1
first 2024
Submissions
1
2024 → 2024
Eligible population
Adult patients aged 18 years or older with hereditary transthyretin amyloidosis confirmed by genetic testing, with stage 1 or stage 2 polyneuropathy (PND score I, II, IIIA, or IIIB), who have not undergone a liver transplant or received previous vutrisiran treatment, and do not exhibit heart failure symptoms (NYHA class III or IV).
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
mNIS+7 composite score
Pivotal trial size
164 patients
Key trials
HELIOS-A, APOLLO
Comparator
patisiran
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis; no ICER calculated by design.