PBAC's latest decision on Lumasiran: Recommended with restriction (2025). Considered for Primary hyperoxaluria type 1 (PH1) in patients with elevated urinary oxalate levels or clinical symptoms indicative of hyperoxaluria, as an addition to best supportive care.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Patients with primary hyperoxaluria type 1 confirmed by genetic testing, with either urinary oxalate ≥0.70 mmol/24 h/1.73 m² or urinary oxalate:creatinine ratio greater than upper limit of normal, or clinical symptoms such as nephrocalcinosis, renal stones, renal impairment, or systemic oxalosis; excluding those with prior liver transplant.
Therapy area
Rare disease
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Surrogate
Key trials
ILLUMINATE-A, ILLUMINATE-B
Comparator
best supportive care (BSC)
Economic model
CUA
ICER note
ICER not stated in the public summary document; appears to be commercially sensitive or redacted.