First combination therapy TGA approved for rare brain cancer

Pharma News: Rare Cancers Australia (RCA) is celebrating the recent green light from the Therapeutic Goods Administration (TGA) for Novartis’ combination targeted therapy, Tafinlar-Mekinist. This approval is for the treatment of a rare form of cancer mutation, BRAF 600E, prevalent in paediatric low and high-grade glioma patients.

Christine Cockburn, CEO of RCA, stated “I’m encouraged by the TGA’s decision to register the Tafinlar + Mekinist therapy, a potentially life-saving drug for the most vulnerable members of our community – our children.”

The therapy has demonstrated remarkable efficacy in slowing tumour growth by impeding signals associated with the BRAF and MEK kinases, pivotal in the development of various cancers.

Ms Cockburn remarked “Too often we see people with rare and less common cancers not receiving the same level of support, or access to the same treatment options, as those with more common cancers. And they pay for that inequity with their lives.”

She further highlighted the significance of the TGA approval, considering the prevalence of gliomas in the paediatric population. Gliomas, the most common paediatric central nervous system tumour type, represent approximately half of all paediatric brain cancers.

“To demonstrate how rare this mutation is and how targeted this therapy you just have to look at the numbers,” Ms Cockburn explained. In Australia, around 140 children are diagnosed with low-grade glioma annually, with 10-20 having the BRAF V600E mutation. Meanwhile, approximately 60 children are diagnosed with high-grade glioma each year, with 5-10 testing positive for the BRAF V600E mutation.

In children as young as 1, Tafinlar-Mekinist cut the risk of progression or death by 69% over chemotherapy when given as first systemic treatment for BRAF V600 paediatric low-grade glioma. In a single-arm cohort of children with relapsed or refractory BRAF V600 high-grade gliomas, the Novartis combo showed a response rate of 56.1%.

Disturbingly, brain cancer accounts for 40% of cancer deaths among children in Australia, and high-grade glioma is the leading cause of these fatalities.

“These approvals make Tafinlar + Mekinist the first and only approved combination targeted therapy to treat paediatric patients with the BRAF V600E mutation,” Ms Cockburn highlighted.

As science progresses in the genomics space, Ms Cockburn envisions a future with more targeted therapies for rare and less common cancers registered in Australia and promptly accessible to patients through the Pharmaceutical Benefits Scheme (PBS).

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