PBAC's latest decision on Nusinersen: Recommended with restriction (2023). Considered for Initial treatment of pre-symptomatic spinal muscular atrophy (SMA) in individuals with SMN1 deletion or mutation and 3 copies of the SMN2 gene, aged less than 18 years.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
9
first 2017
Submissions
9
2017 → 2023
Eligible population
Pre-symptomatic individuals with genetically confirmed 5q SMA (SMN1 deletion or mutation) with 3 copies of the SMN2 gene, aged less than 18 years, untreated with gene therapy.
Therapy area
Neurology
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
Time to death or respiratory intervention
Comparator
nusinersen upon symptom onset (symptomatic treatment)
Economic model
CUA
ICER note
ICER values are redacted (commercial-in-confidence). The PSD states the economic model was updated but does not publish numeric ICER figures in the public document.
Risk sharing
Risk-sharing arrangement in place — A rebate was proposed for individuals with pre-symptomatic SMA with 3 SMN2 copies, aligned with the special pricing arrangement (SPA) currently applied in the symptomatic setting.
Submission history
Nov 2017: Recommended with restriction · Authority Required
Mar 2018: Recommended with restriction · Authority Required
Jul 2018: Recommended with restriction · Authority Required
Jul 2019: Not recommended · Authority Required
Jul 2020: Recommended with restriction · Authority Required
Nov 2020: Deferred · Authority Required
Jul 2021: Recommended with restriction · Authority Required
Mar 2022: Recommended with restriction · Authority Required
Jul 2023: Recommended with restriction · Authority Required