The latest round of PBAC deliberations has delivered several wins for neurological care, with new treatment options recommended for generalised myasthenia gravis, Lennox-Gastaut syndrome and spinal muscular atrophy. The committee also supported PBS listing of the first therapy for Friedreich’s ataxia.

Subcutaneous efgartigimod approved for gMG

The Pharmaceutical Benefits Advisory Committee recommended a Section 100 listing for the new subcutaneous formulation of efgartigimod alfa (Vyvgart®) for adults with anti-AChR antibody-positive generalised myasthenia gravis.

The PBAC accepted that the subcutaneous form was non-inferior in effectiveness and safety to intravenous efgartigimod. It also supported the proposed equi-effective dose of 1,000 mg SC as equivalent to 961 mg IV, and advised the medicine be priced in line with the four gMG therapies recommended at its March 2025 meeting and folded into the same risk-sharing arrangement.

Submissions from clinicians and advocacy groups emphasised the heavy and fluctuating symptom burden of gMG, with the PBAC noting the potential for improved access, reduced treatment complexity and meaningful gains in quality of life.

Fenfluramine supported for Lennox-Gastaut syndrome

Fenfluramine (Fintepla®) was recommended as add-on therapy for people with Lennox-Gastaut syndrome who have not responded to at least two anti-seizure medicines.

The PBAC acknowledged the severe and lifelong nature of LGS, the high caregiver burden, and the limited effectiveness of existing PBS-subsidised options, including cannabidiol.

While the Committee said clinical evidence remains “uncertain”, it accepted that fenfluramine was as effective as cannabidiol, but flagged important safety concerns, including appetite suppression, weight loss, and a “known risk of serious cardiac events”. Long-term cardiac safety was considered unresolved.

The PBAC advised that fenfluramine carry similar restrictions to cannabidiol, including cardiac monitoring, and concluded the incremental cost of listing was likely small. It recommended including the therapy within the cannabidiol risk-sharing framework without altering expenditure caps.

Zolgensma® access expanded for paediatric SMA patients

Access to onasemnogene abeparvovec (Zolgensma®) will broaden under a PBAC recommendation to extend PBS eligibility to children weighing up to 21 kg.

The change applies to:

• symptomatic Type I–IIIa SMA (≤21 kg)
• pre-symptomatic SMA with 1–3 SMN2 copies treated before 36 month
• untreated pre-symptomatic SMA with 1–3 SMN2 copies up to 36 months of age

Expert input advised against restricting symptomatic treatment to Type I alone, warning this risked excluding children with later symptom onset.

Although evidence in older children is limited, the PBAC considered Zolgensma as effective and as safe in those older than nine months as in younger cohorts, while reiterating that “earlier initiation of disease-modifying therapy remains the clinical preference”.

First therapy for Friedreich’s ataxia recommended

Omaveloxolone (Skyclarys®) was recommended for adults and adolescents aged 16 years and over with Friedreich’s ataxia, following a resubmission supported strongly by clinicians and patient groups.

The PBAC considered the therapy “more effective than best supportive care” at slowing functional decline, though the expected benefit was characterised as modest. It also recognised safety concerns, including cardiotoxicity and elevated liver enzymes.

However, the Committee rejected the sponsor’s “overestimated duration-of-benefit” assumptions and said the medicine would be “cost-effective only with a substantial price reduction”, paired with a risk-sharing arrangement to manage use outside the proposed restriction criteria.

Tablet formulation of risdiplam approved

The PBAC also backed PBS listing of risdiplam 5 mg tablets (Evrysdi®) for the treatment of patients with spinal muscular atrophy, finding the formulation non-inferior to the oral solution in both safety and efficacy. Consumer input highlighted greater convenience, portability and dosing flexibility for some families.