PBS backs first targeted therapy for rare brain cancer in over two decades

Australians living with a rare, incurable form of brain cancer will gain subsidised access to the first targeted therapy for the disease in more than two decades, following the listing of Servier’s Voranigo (vorasidenib) on the Pharmaceutical Benefits Scheme (PBS).

From 1 August, the first-in-class oral isocitrate dehydrogenase (IDH) 1/2 dual inhibitor will be available through the PBS for adults and adolescents aged 12 years and older with grade 2 IDH-mutant astrocytoma or oligodendroglioma who have not previously received systemic therapy or radiotherapy.

Servier obtained Voranigo through its US$2 billion acquisition of Agios Pharmaceuticals’ oncology business in 2021.

“For people living with IDH-mutant glioma, managing the disease involves making complex treatment decisions over many years. Caring for these patients often involves balancing long-term disease control with preserving neurological function and quality of life,” said Associate Professor Jim Whittle, Medical Oncologist at Peter MacCallum Cancer Centre and Co-Director of Research Strategy, The Brain Cancer Centre at WEHI. “Access to vorasidenib through the PBS provides clinicians with a targeted treatment option, adding another tool to the toolbox and supporting greater choice when discussing treatment options with patients.”

Gliomas are the most common primary malignant brain tumours in adults, with IDH1 or IDH2 mutations present in nearly all grade 2 diffuse gliomas. An estimated 3,230 Australians are living with an IDH-mutant glioma, while around 135 new cases are diagnosed each year. Typically affecting people between the ages of 20 and 45, the slow-growing but incurable disease can progress over many years, leaving patients to navigate ongoing uncertainty, seizures, cognitive changes, physical disability and loss of independence during key stages of their lives, including building careers, raising families and planning for the future.

Until now, treatment has largely relied on surgery, active monitoring and, where required, radiotherapy or chemotherapy to slow disease progression.

Results from the phase 3 INDIGO trial demonstrated that Voranigo reduced the risk of tumour progression or death by 61% compared with placebo in patients with residual or recurrent grade 2 IDH-mutant glioma who had received no previous treatment other than surgery. The therapy also extended median progression-free survival (PFS) from 11.1 months to 27.7 months.

“Behind every IDH-mutant glioma diagnosis is a person, a family and a future forever changed. For our community, today’s PBS listing is a significant milestone after years of advocacy to improve access to new treatment options for Australians living with this rare brain cancer,” said Craig Cardinal, Chair of the Australian Brain Tumour Collaborative (ABTC).

“Access to treatment is only one part of the journey. People living with brain cancer and their families also need coordinated multidisciplinary care, psychosocial support and practical services throughout every stage of their diagnosis.” Cardinal added.

Mélanie Chamaux, General Manager of Servier Australia and New Zealand, said “We are incredibly proud of the PBS listing of Voranigo, which marks an important milestone for Australians living with IDH-mutant glioma. The listing represents a collective achievement of the healthcare system recognising therapeutic progress and the meaningful difference innovative medicines can make for patients, their families and the broader community. We hope this milestone brings renewed confidence and optimism to those facing the challenges of this disease.”

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