OmaveloxoloneSkyclarys
Treatment of Friedreich's ataxia in people aged 16 years and older.
Decisions on record
- Meeting Nov 2025 Recommended Friedreich's ataxia in adults and adolescents aged 16 years and older
- Meeting Mar 2025 Not recommended Friedreich's ataxia in adults and adolescents aged ≥16 years
Access path
- TGA registered · Skyclarys
TGA label narrower than the PBS population
- Mar 2025Not recommended
vs best supportive care
- ↻ resubmittedNov 2025Recommended · restricted
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the General Schedule, Authority Required (telephone/online) listing of omaveloxolone on the PBS for the treatment of Friedreich's ataxia (FA) in adults and adolescents aged 16 years and over. The PBAC noted the high unmet need for treatments for FA. The PBAC considered that omaveloxolone was likely to be clinically superior compared to best supportive care (BSC).PSD · Nov 2025
7.2 The PBAC noted the consumer input received with this resubmission and with the original submission in March 2025 which strongly supported the submission. The PBAC noted that the input highlighted the high need for treatments for FA, the debilitating effect of symptoms of FA on patients and the burden on patients and caregivers.PSD · Nov 2025
6.50 The resubmission presented an economic evaluation that compared treatment with omaveloxolone + BSC to BSC only, in patients diagnosed with FA and aged over 16 years. The type of economic evaluation presented was a cost-utility analysis and the key components are summarised in Table 11. 23 OFFICIALPSD · Nov 2025
OFFICIAL Public Summary Document - November 2025 PBAC Meeting Table 11: Summary of model structure, key inputs and rationale Component Summary Four cohorts were modelled depending on the age of symptom onset (≤7 years, 8 – 14 years, 15 – 24 Cohorts modelled years and > 24 years). Outcomes Life years and QALYs.PSD · Nov 2025
Source: Table 1.1, p8 of the resubmission Blue shading indicates information previously considered by the PBAC.PSD · Nov 2025
6.45 The resubmission again described omaveloxolone as superior in terms of effectiveness and inferior in terms of safety compared to BSC. 21 OFFICIALPSD · Nov 2025
6.6 The PBAC noted that advice received from the National Paediatric Medicines Forum which, noting the high unmet need for disease-modifying treatments in FA, supported the resubmission.PSD · Nov 2025
6.7 The PBAC again noted the input from health professionals received in March 2025 from neurologists, clinical researchers/investigators, physiotherapists and an occupational therapist. The health professionals described the significant impact of this progressive neurogenerative condition on quality of life, stating that FA slowly reduces the capacity for people to undertake daily tasks, walk, talk and swallow food.PSD · Nov 2025
Cost-effectiveness
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
The PBAC advised that omaveloxolone was not cost-effective with an incremental cost-effectiveness ratio (ICER) of > $1,055,000 per quality adjusted life year (QALY) gained. PBAC · 2025
Decision context
PopulationPeople aged 16 years and older with Friedreich's ataxia confirmed by FXN gene mutation, with mFARS score of 20 to 80
Risk sharingA cost-sharing rebate mechanism between the sponsor and Government was proposed for expenditures exceeding expected expenditure caps
Why it was knocked back
- 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
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Recommended · restricted | best supportive care | — | RCT · Change in modified Friedreich's ataxia rating scale (mFARS) score at week 48 |
| Mar 2025 | Not recommended | best supportive care | — | RCT · Change in modified Friedreich's ataxia rating scale (mFARS) score at week 48; Safety outcomes |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| MOXIe Part 2 | Ph 3 | 255 | Part 1 RCT: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified F… | recruiting |
Consumer voice
Consumer input from patients with Friedreich Ataxia (FA) and their caregivers described the progressive, debilitating nature of the condition affecting mobility, speech and coordination, and emphasized the importance of treatments that delay disease progression. Comments highlighted that omaveloxolone, while not a cure, has potential to preserve functionality, reduce symptoms, and delay serious co
Input described the impacts of this condition, which consist of progressive symptoms affecting walking, balance, speech and coordination, reducing people's ability to undertake daily tasks and quality of life. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with FXN gene mutation confirmation and mFARS score 20–80; TGA label has no such biomarker or severity constraints.