PBAC's latest decision on Onasemnogene abeparvovec: Not recommended (2023). Considered for Pre-symptomatic treatment of babies with spinal muscular atrophy (SMA) and 3 copies of the Survival Motor Neuron 2 (SMN2) gene.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
3
first 2020
Submissions
3
2020 → 2023
Eligible population
Paediatric patients less than 9 months of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 3 copies of the SMN2 gene.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
Surrogate
Pivotal trial size
25 patients
Key trials
SPR1NT
Comparator
onasemnogene abeparvovec in patients with 1-2 copies of SMN2 gene
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis presented; no ICER calculated as the submission used a cost-minimisation approach comparing ONA in 3 SMN2 copy patients versus ONA in 2 SMN2 copy patients.
Risk sharing
Risk-sharing arrangement in place — Outcomes-based Risk Sharing Agreement (RSA) was mentioned in context of the previous September 2021 recommendation for the 1-2 SMN2 copy population.
Why PBAC said no
Reasons cited in the latest PSD: Unclear incremental clinical benefit for patients with 3 SMN2 copies compared to symptomatic treatment with disease-modifying therapies; limited clinical data; cost-minimisation comparison against ONA in 2 SMN2 copy patients was inconsistent with PBAC's previous view; lack of long-term follow-up data; uncertainty regarding subsequent therapy requirements; assumed zero need for follow-on treatment not adequately supported.