DFMO approved for use in Australia

Norgine has received regulatory approval from the TGA to distribute DFMO (eflornithine), a life-changing treatment for high-risk neuroblastoma, in Australia. We are the first country outside of the USA to receive such approval.

This milestone marks a major step forward in improving outcomes for children diagnosed with this terrible disease, and will make a significant difference to families across the country.

DFMO was approved by the FDA in the USA in December 2023. This was based on clinical trial data showing it reduced the rate of relapse by an impressive 50%. While it has been available here in Australia (initially through the Federal Government providing interim funding, and then subsequently through the drug distributor supplying for free via an Expanded Access Program), this result means the availability of DFMO is now secured for the future.

Neuroblastoma Australia requested the interim funding for DFMO from the Federal Government and was successful thanks to incredible collaboration and relentless advocacy.

We would like to sincerely thank:

First and foremost, the families who courageously shared their stories—particularly the families of Harry and Zai, whose powerful testimonies underlined the vital and urgent need to access DFMO in Australia

Members of both major political parties who supported the cause, especially the Health Minister Mark Butler MP and Shadow Health Minister, Senator Anne Ruston

Brilliant scientists at Children’s Cancer Institute for their expertise and commitment

The dedicated team at CT Group for their strategic guidance and advocacy

The Pharmaceutical Benefits Advisory Committee (PBAC) has also approved access to DFMO via the Pharmaceutical Benefits Scheme (PBS) which will means families can access the treatment at minimal cost (exact subsidy amount to be confirmed). Read Norgine’s (provider of ethlornifine in Australia) media statement:

Media Release

Our final thank you is to everyone who has supported our small charity. Whether you’ve made a donation, signed-up for Run2Cure, held a community fundraiser, or simply shared a social media post – this result would not have been possible without your help.

We have always believed in the importance of our role as the only charity in Australia to focus on improving outcomes for children with neuroblastoma. This result is a clear demonstration of our ability to achieve real and meaningful change for the young children diagnosed with this devastating childhood cancer.

Kind regards,

Lucy

Why we support DFMO

DFMO was shown to nearly halve the risk of relapse based on a clinical trial led by Dr Giselle Sholler and the Beat Childhood Cancer Research Consortium in collaboration with US WorldMeds, published in Journal of Clinical Oncology on Oct. 23. According to Dr. Sholler, the evidence provided by this study led directly to FDA approval for DFMO. Her study showed four-year event free survival was 84% versus 72% in the DFMO and NO-DFMO groups, respectively. Overall survival was 96% versus 84% respectively.

Please contact Lucy at Lucy.Jones@nb.org.au if you would like more information.