Australian children with neuroblastoma given free access to $500,000 cancer drug DFMO

Australian children with neuroblastoma given free access to $500,000 cancer drug DFMO
  • PublishedJuly 10, 2024

Australian children and young adults with neuroblastoma, a cancer that affects the nervous system, will be given a desperately needed lifeline to access medicine previously only available by travelling overseas.

There are about 20 Australian children with high-risk neuroblastoma, which most commonly affects babies and children under 5 years old.

Last month, the ABC reported the story of two-year-old Luna, who had endured five rounds of chemotherapy, surgeries, a bone marrow transplant and immunotherapy as part of her treatment for the rare cancer.

Luna’s family and others had pleaded for access to a promising medication that could help to treat the cancer, but which was exorbitantly expensive and not yet approved in Australia.

The medication, known as DFMO (difluoromethylornithine), was approved in the United States at the end of last year, but because it is not available in Australia, some families were spending more than $500,000 to pursue treatment in the US.

Other children missed out because they couldn’t afford treatment or were too sick to travel.

Health Minister Mark Butler announced on Wednesday morning after engaging with the drug maker Norgine over several weeks, the government had secured an undertaking that the company would provide DFMO to Australian patients for free, while it pursued local approvals for the medicine to be listed on Australia’s Pharmaceutical Benefits Scheme.

Mr Butler said until that compassionate access scheme is established, the government would fund major paediatric hospitals to provide DFMO to children for free.

“We understand that DFMO offers the only hope to some patients who are desperately ill from neuroblastoma,” Mr Butler said.

“This one-off funding ensures that they can get this new and promising treatment, without the huge price tag, while proper approval processes are followed.”

About half of children diagnosed with neuroblastoma do not survive, and more than half of those who are successfully treated have recurrences that lower survival rates to just 5 per cent.

DFMO was found in trials to reduce the risk of neuroblastoma recurrence by 50 per cent, helping about three-quarters of children diagnosed to survive the disease.


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