PBAC's latest decision on Cerliponase alfa: Recommended with restriction (2018). Considered for Treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare neurodegenerative genetic condition caused by deficiency of the enzyme TPP1.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2018
Submissions
1
2018 → 2018
Eligible population
Patients with confirmed neuronal ceroid lipofuscinosis type 2 disease, including those with early to moderate symptoms (ML score 3-6) aged 3-15 years, with stable seizures.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
ORR
Pivotal trial size
24 patients
Key trials
Study 201/202, Study 901
Comparator
standard care
Economic model
CUA
ICER note
ICER values are redacted in the public document; economic model based on life years gained and cost-utility analysis but specific ICER figures not stated.