Government breaks barriers to access childhood brain cancer therapy

Pharma News: In combating the rare and highly aggressive childhood brain cancer, Diffuse Intrinsic Pontine Glioma (DIPG), the Australian Government has successfully secured access to an experimental medicine developed by Chimerix, ONC201 (dordaviprone).

The medicine will now be available to eligible Australian patients through an Expanded Access Program, offering hope to those grappling with the challenges posed by DIPG. In addition to this access, the government has allocated up to $20 million over seven years through the Medical Research Future Fund (MRFF). This funding will support crucial clinical trials and essential research aimed at discovering novel treatments for childhood brain cancers.

Health Minister Mark Butler MP underscored the government’s commitment to ensuring access to vital medicines, stating, “Making sure all Australians have access to important and lifesaving medicines is an Albanese Government priority. I know Eve Daher’s family faced an uphill battle to get ONC201. This access to ONC201 is a great result for Eve and potentially other young Australians affected by DIPG.”

Despite an overall childhood cancer survival rate exceeding 80%, brain cancers remain the most prevalent solid tumors in children, claiming more lives than any other cancer type. DIPG, in particular, stands out as an exceptionally challenging adversary, with only 1 in 10 children surviving for two years post-diagnosis, and less than 1 in 100 surviving for five years.

The breakthrough access to ONC201 comes following Minister Butler’s proactive engagement with the US pharmaceutical company, Chimerix. Minister Butler sought access to the experimental medicine through both Chimerix’s Expanded Access Program and the Therapeutic Goods Administration (TGA)-administered Special Access Program for unapproved medicines.

In response to Minister Butler’s advocacy, Chimerix has not only granted access to ONC201 but has also expanded its medical scope to include certain patients with H3 K27M mutation and/or midline gliomas, encompassing those already undergoing ONC201 treatment. Moreover, the TGA is actively providing essential information to Chimerix regarding Australia’s requirements for potential future registration of the medicine in the country.

As part of this initiative, the establishment of the National Childhood Brain Cancer Clinical Trial Consortium will further strengthen the collaborative effort against DIPG. The Consortium aims to foster international cooperation and interdisciplinary collaboration within the research sector. By promoting new research approaches to treatment development and enhancing clinical care, the Consortium seeks to expedite the assessment of safety and efficacy of current and emerging treatments, which is aligned with the ongoing HTA review.

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