PBAC's latest decision on Tofersen: Recommended with restriction (2025). Considered for Adjunct treatment to best supportive care with or without riluzole in patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene pathogenic variant.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Adults with amyotrophic lateral sclerosis (ALS) who have a confirmed pathogenic or likely pathogenic superoxide dismutase 1 (SOD1) gene variant, have not undergone tracheostomy or received permanent non-invasive ventilation, and have not experienced respiratory failure.
Therapy area
Neurology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Other
Key trials
VALOR (Study 101, Part C), VALOR open label extension (Study 102)
Comparator
best supportive care (BSC) with or without riluzole
Economic model
CUA
ICER note
ICER values are redacted (commercial-in-confidence) in the public summary document.