TOFERSEN

The PBAC recommended listing tofersen (Qalsody®) on the PBS for the treatment of patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dimutase 1 (SOD1) gene pathogenic variant.
The PBAC recalled receiving input from individuals, health care professionals and organisations in November 2025 with the original submission. It noted the high unmet need for targeted treatments for this rare subtype of ALS. The PBAC acknowledged the progressive nature of the disease and that any slowing of disease progression or retention of functional capacities was a crucial factor in improving patient quality of life.  
 The PBAC recalled that in November 2025 it had considered that, although not demonstrated statistically in the trial, based on biomarkers (plasma neurofilament light chain, an indicator of neurological disease progression), tofersen was likely more effective than best supportive care at slowing the progression of SOD1-ALS and increasing overall survival. However, the PBAC noted that the effect of tofersen in preventing loss of physical function would likely be modest. The PBAC considered that given the progressive nature of SOD1-ALS, stabilisation of disease would still be beneficial for patients, their families and their carers. The PBAC also previously noted that tofersen was less safe than best supportive care, with side effects mainly associated with its administration via lumbar puncture.
The PBAC considered that the cost requested by the sponsor in the resubmission was acceptable. The PBAC also considered that the estimates of eligible patients and associated costs presented in the resubmission were reasonable with minor changes. The PBAC considered that the risk sharing arrangement proposed by the sponsor would mitigate the risk associated with the uncertain long-term benefits of tofersen and the uncertain duration of therapy.
The PBAC advised that the listing would require changes to the restrictions for the PBS-listed medicine edaravone to prevent combined use of edaravone and tofersen.