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
5.1 The PBAC recommended tofersen for the treatment of patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene pathogenic variant. The PBAC noted the high unmet need for targeted treatments for this rare subtype of ALS.PSD · Mar 2026
OFFICIAL Public Summary Document – March 2026 PBAC Meeting that the cost-effectiveness of tofersen was uncertain due to the limited data informing the economic model, but that in the context of this life-limiting disease in a rare sub-group of ALS patients, and considering the expected level of improvement in quality of life and survival, the tofersen was cost-effective at the price proposed in the resubmission, in the context of the proposed Risk Sharing …PSD · Mar 2026
4.12 The resubmission presented an updated economic model which included the revised prices of tofersen (see paragraph 3.4 and Table 5). There were no other changes applied to the economic model in the resubmission.PSD · Mar 2026
Table 5: Updated effective DPMQs of tofersen (assuming 50% of tofersen use would be in private hospitals) Treatment phase Description Effective EMP per vial Effective DPMQ Model cycles (weighted)a applied Initiation phase Tofersen 100 mg vial $ $ 1-6 redacted content redacted content First continuation phase Tofersen 100 mg vial $ $ 7-13 redacted content redacted content Response phase Tofersen 100 mg vial $ $ 14+ redacted content redacted content Source: …PSD · Mar 2026
compared to best supportive care with or without riluzole. Source: Table 1, p1 November 2025 minutes.PSD · Mar 2026
4.11 The resubmission did not provide new clinical claims, rather it appeared to accept the PBAC’s considerations in November 2025 that:PSD · Mar 2026
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 | $955k–1.05m | 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.