TofersenQalsody
Treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.
Decisions on record
- Meeting Mar 2026 Recommended Amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) gene pathogenic variant
- Meeting Nov 2025 Not recommended Amyotrophic lateral sclerosis with superoxide dismutase 1 gene pathogenic variant, no respiratory failure
Access path
- TGA registered · Qalsody
TGA label narrower than the PBS population
- Nov 2025Not recommended
vs best supportive care (BSC) with or without riluzole
- ↻ resubmittedMar 2026Recommended · restricted
Comparator changed: best supportive care (BSC) with or without riluzole → placebo, as a…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC did not recommend tofersen for the treatment of patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene pathogenic variant. The PBAC acknowledged the high unmet need for targeted treatments for this rare subtype of ALS.PSD · Nov 2025
OFFICIAL Public Summary Document – November 2025 PBAC Meeting (ICER) was very high and that the economic model was not sufficiently reliable for decision-making due to the limited clinical data informing the model. The PBAC also noted that the cost per patient per year of tofersen was substantially higher than that for previously recommended treatments for rare diseases funded on the PBS with benefits which are likely similar in terms of clinical impact.PSD · Nov 2025
6.39 The submission presented a Markov cohort, cost-utility analysis comparing tofersen plus BSC with or without riluzole (tofersen arm) to BSC with or without riluzole alone (BSC arm) for SOD1-ALS, based on the clinical claim of superior effectiveness for tofersen plus BSC versus BSC.PSD · Nov 2025
OFFICIAL Public Summary Document – November 2025 PBAC Meeting latest available DCOs (16 Jan 2022 for MiToS stage progression, 28 Feb 2023 for survival), even when controlling for treatment switching. Additionally, as all patients in VALOR OLE received tofersen, no comparative data versus placebo were available beyond 28 weeks.PSD · Nov 2025
compared to best supportive care with or without riluzole. Source: Table 1.1, p3 of the submission.PSD · Nov 2025
AE=adverse event; ALS=amyotrophic lateral sclerosis; CSF=cerebrospinal fluid; NfL=neurofilament light chain; SAE=serious adverse event; SOD1=superoxide dismutase 1; SVC=slow vital capacity;PSD · Nov 2025
6.2 The PBAC noted and welcomed the input from individuals (33), health care professionals (1) and organisations (1) via the Office of Health Technology Assessment Consultation Hub. The comments from individuals described the symptoms of SOD1- ALS, the progressive nature of the disease and noted that current standard of care focused on managing symptoms of disease.PSD · Nov 2025
The ESC noted that input indicated that consumers considered the availability of a genetically targeted treatment with potentially disease-modifying effects was a significant advancement, providing hope, even for patients with subtypes other than SOD1-ALS.PSD · Nov 2025
Cost-effectiveness
ICER values are commercially sensitive and redacted in the public document. Redacted ranges indicate ICER of $1 per QALY (reduced from $2 per QALY in November 2025 submission).
The PBAC considered that the ICER was very high and unreliable in the November 2025 submission, and that the value proposition was difficult to assess given the uncertainty in the ICER which reflected the limited clinical data, the small number of patients included in the studies and the individualised nature of ALS-SOD1 progression. PBAC · 2026
Decision context
PopulationAdults with amyotrophic lateral sclerosis who have a superoxide dismutase 1 (SOD1) gene pathogenic variant, without prior tracheostomy or respiratory failure, receiving best supportive care with or without riluzole.
Risk sharingRisk Sharing Arrangement proposed with a percentage rebate on expenditure over the Deed term, with higher rebate levels implemented in a pay-for-performance framework tied to response to treatment (defined as no more than 20% decline from baseline ALSFRS-R at 12 months).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | placebo, as a proxy for best supportive care (BSC) with or without riluzole | — | RCT · ALSFRS-R |
| Nov 2025 | Not recommended | best supportive care (BSC) with or without riluzole | — | RCT · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| VALOR (Phase 3, Study 101, Part C) | Ph 3 | 158 | Parts B and C: Percentage of Participants with Emergence of Clinically Manifest ALS Within… | active not recruiting |
Consumer voice
Consumer input from 33 individuals, 1 health professional, and 1 organisation (Motor Neuron Disease Australia) described SOD1-ALS as progressive and symptomatic, welcoming tofersen as the first gene-targeted therapy offering disease modification and hope. Comments noted some adverse events from lumbar puncture administration but emphasized quality of life improvements, though access barriers and h
tofersen was the first gene-targeted therapy which delayed disease and symptom progression and presented the ability to maintain independence Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line, excludes tracheostomy/permanent ventilation/respiratory failure; TGA label has no such clinical restrictions.