PBAC greenlights novel cancer and rare disease treatments

The Pharmaceutical Benefits Advisory Committee (PBAC) has delivered a wave of positive recommendations following its March meeting, paving the way for access to treatments in multiple myeloma, breast cancer, neuroblastoma, lung cancer, and a range of rare diseases.

Johnson & Johnson Innovative Medicine’s Darzalex (daratumumab), a CD38-directed monoclonal antibody, was recommended for listing on the Pharmaceutical Benefits Scheme (PBS) as a first-line treatment for multiple myeloma patients who are ineligible for stem-cell transplantation. This therapy, in combination with lenalidomide and dexamethasone, would be available to around 1,500 newly diagnosed myeloma patients annually.

Joana De Castro, Managing Director of Johnson & Johnson Innovative Medicine Australia & New Zealand, said “This funding recommendation brings Australia one step closer to joining 38 other countries where Darzalex is reimbursed as a first-line treatment. We hope that Australians with newly diagnosed multiple myeloma will soon have access to the same first-line treatment as other myeloma patients around the world.”

The PBAC also acknowledged the significant number of treatments already available for multiple myeloma on the PBS and recommended that the Department of Health conduct a utilisation analysis to better understand treatment pathways and durations for existing therapies.

Breast Cancer Network Australia (BCNA) has welcomed PBAC’s decision to recommend AstraZeneca’s Truqap (capivasertib) in combination with hormone blocking treatment fulvestranta for HR+ / HER2- locally advanced or metastatic breast cancer, following recurrence or progression on or after endocrine therapy.

Nurse Practitioner, Jenny Gilchrist, said “Capivasertib represents a significant advancement for eligible metastatic breast cancer patients, doubling progression-free survival when combined with fulvestrant.”

Vicki Durston, BCNA Director of Policy, Advocacy and Support Services, said this has been a long-awaited outcome.

“It is imperative people with metastatic breast cancer, a growing group with significant unmet needs, have timely access to new and innovative therapies,” stated Durston.

Meanwhile, for Australians impacted by high-risk neuroblastoma, the PBAC’s recommendation for Norgine’s Ifinwil (eflornithine) offers a critical breakthrough. Fast-tracked by the TGA under Project Orbis, the positive recommendation for PBS listing follows strong advocacy from the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG), Neuroblastoma Australia, and political pressure from Shadow Health Minister Senator Anne Ruston, who raised serious concerns over the delayed funding, highlighting the devastatingly low survival rates of just 10–20% in relapsed cases.

Lung cancer patients also received good news, with Novartis’ Mekinist (dabrafenib) plus trametinib recommended for adults with BRAF V600E mutation-positive metastatic non-small cell lung cancer (NSCLC). Despite uncertainties in the indirect comparisons presented, the PBAC concluded that the Mekinist combination would likely deliver comparable health outcomes to MSD’s Keytruda (pembrolizumab) plus chemotherapy, deeming it cost-effective if priced competitively against the Keytruda regimen.

In rare liver disease, Ipsen’s Iqirvo (elafibranor) was recommended for PBS listing to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA). This first-in-class PPAR oral therapy competes with Chiesi’s Ocaliva (obeticholic acid), already listed on the PBS. The PBAC noted that a price premium for Iqirvo would be justified, given its potential to reduce PBC-related pruritus compared to OCA.

For patients living with generalised myasthenia gravis (gMG), the PBAC reviewed therapies from UCB – Rystiggo (rozanolixizumab), Zilbrysq (zilucoplan), argenx – Vyvgart (efgartigimod alfa), and Alexion – Ultomiris (ravulizumab). It concluded that none of these therapies demonstrated superior efficacy or safety compared to intravenous immunoglobulin or plasma exchange. However, they were recommended for PBS listing based on cost comparison with intravenous immunoglobulin, supported by cost-per-responder analysis against placebo.

The PBAC also considered funding for two proposed extensions to existing cystic fibrosis treatments, both of which were recommended for listing. Vertex’s Trikafta, now extended to include children aged 2 years and older with a responsive CFTR gene mutation, would provide access to CFTR modulator therapy for 45 individuals. Approximately 150 current users of Symdeko or Kalydeco may also qualify for transitioning to Trikafta. Additionally, Kalydeco has been recommended for extension to include infants aged 1 to 4 months, offering early intervention to around 10 babies annually.

The final hurdle remains: pricing negotiations between the government and pharmaceutical companies before the PBS listings are officially secured.

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