PBAC's latest decision on Omaveloxolone: Not recommended (2025). Considered for Treatment of Friedreich's ataxia in people aged 16 years and older.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
—
Submissions
2
first 2025
Submissions
2
2025 → 2025
Eligible population
People aged 16 years and older with Friedreich's ataxia confirmed by FXN gene mutation, with mFARS score of 20 to 80
Therapy area
Rare disease
Line of therapy
Not applicable
Evidence base
RCT
Primary endpoint
Change in modified Friedreich's ataxia rating scale (mFARS) score at week 48
Key trials
MOXIe Part 2
Comparator
best supportive care
Economic model
CUA
ICER note
ICER stated as >$1,055,000 per QALY gained in both March 2025 and November 2025 submissions; exact upper bound not specified, remains above cost-effectiveness threshold
Risk sharing
Risk-sharing arrangement in place — A cost-sharing rebate mechanism between the sponsor and Government was proposed for expenditures exceeding expected expenditure caps
Why PBAC said no
Reasons cited in the latest PSD: ICER >$1,055,000 per QALY gained remains outside range usually considered cost-effective, uncertainty in magnitude and duration of improvement particularly in patients with different disease severity, treatment effect modelling concerns
Submission history
Mar 2025: Recommended with restriction · Authority Required