PBAC's latest decision on Patisiran: Recommended with restriction (2023). Considered for Treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
1
first 2023
Submissions
1
2023 → 2023
Eligible population
Adult patients with hereditary transthyretin-mediated amyloidosis confirmed by genetic testing with stage 1 or stage 2 polyneuropathy (FAP stage 1 or 2, or PND score I, II, IIIA, or IIIB), treated by a neurologist or consultant physician with experience in amyloid disorder management.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
mNIS+7 (modified Neuropathy Impairment Score +7)
Pivotal trial size
360 patients
Key trials
APOLLO, APOLLO-B
Comparator
best supportive care (BSC)
Economic model
CUA
ICER note
ICER value(s) not stated in the provided text; document appears incomplete at section 5.2.