VORASIDENIB

The PBAC recommended the listing of vorasidenib for the treatment of isocitrate dehydrogenase-mutant (IDH) astrocytoma or oligodendroglioma. The PBAC welcomed the strong support from individuals, health care professionals and organisations for this resubmission. The PBAC maintained its previous view that there is a high unmet clinical need for effective treatments for astrocytoma and oligodendroglioma, noting that there are currently no other effective treatments available for patients not in immediate need of chemotherapy/radiotherapy. The PBAC recalled it had noted that as the disease progresses patients experience headaches, nausea/vomiting, seizures, drowsiness, visual disturbance, speech/language problems, sensory loss, motor deficits and changes in cognitive and/or functional ability. These symptoms have a significant impact on quality of life, including inability to work, drive, remain independent, and anxiety is associated with surveillance only, knowing that the condition will inevitably progress. The PBAC also acknowledged that chemotherapy/radiotherapy is associated with substantial toxicity and worsening of neurological deficits.

The PBAC accepted vorasidenib was superior to active surveillance in terms of radiological progression (observable changes in medical imagining) and acknowledged that delaying the need for chemotherapy and radiation therapy and the reduction in epileptic seizures are clinically meaningful outcomes that are likely to positively impact on patient quality of life (QoL).

The PBAC considered the additional trial data, revised estimates of benefits and revised estimates of costs provided in the resubmission addressed most of the Committee’s previous concerns with the July 2025 submission. The PBAC considered vorasidenib would be acceptably cost-effective at the reduced price proposed in the resubmission and the revised financial estimates were considered reasonable.

The PBAC advised that amendments to the sponsor’s proposed approach to managing financial risk were required to address the uncertainty of how long patients may stay on treatment, including the possibility that patients may remain on treatment after disease progression. The PBAC advised that the use of vorasidenib should be reviewed once listed on the PBS to confirm whether patient numbers reflect the estimates of use provided in the resubmission.