Media Release, 4 March 2024: Rare Cancers Australia (RCA) has welcomed a recent approval by the Therapeutic Goods Administration (TGA) of the Novartis targeted therapy drug Tafinlar + Mekinist, which has proven to be effective in treating an extremely rare form of cancer mutation, called BRAF 600E, in paediatric low and high grade glioma patients.

While this mutation is rare, Gliomas are the most common paediatric central nervous system tumor type, representing approximately half of all pediatric brain cancers. Research has shown that this therapy may help to slow tumor growth by blocking signals associated with the BRAF and MEK kinases that are implicated in the growth of various types of cancer.

CEO of RCA, Christine Cockburn, stated the approval was a positive step towards equitable access for the innocent Australian children living with the rare mutation.

“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,” said Ms Cockburn.

“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

Child Brain Cancer Patient, image provided by Rare Cancer

that inequity with their lives.”

“As our ability to target rare and less common cancers advances through the scientific development of immunotherapies and genomics, I look forward to more announcements of this nature and the promise of improved outcomes for all people living with cancer”, Ms Cockburn said.

To demonstrate how rare this mutation is and how targeted this therapy you just have to look at the numbers.

In Australia, approximately 140 children are diagnosed with low grade glioma each year, of which 10-20 will have the BRAF V600E mutation, while approximately 60 children will be diagnosed with high grade glioma each year, with 5-10 of those being positive for the BRAF V600E mutation.

Brain cancer accounts for 40% of cancer deaths among children in Australia, and high grade glioma accounts for most of these deaths.

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

“As science continues to advance in the genomics space, we hope to see more targeted therapies for rare and less common cancers registered in Australia and made available to patients on the PBS in a timely manner” she said.


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