Eflornithine has been approved for high-risk neuroblastoma patients who have responded to prior therapy.
Last week Norgine, a specialist pharmaceutical company based in Europe, announced that the TGA had approved the use of eflornithine for the treatment of adult and paediatric patients with high-risk neuroblastoma who had previously responded to multiagent, multimodality therapy.
Eflornithine’s efficacy was demonstrated through a controlled trial comparison, where 90 paediatric patients with high-risk neuroblastoma (who received twice-daily doses for up to two years and were followed for up to seven years) were compared to 270 control patients from a separate high-risk neuroblastoma trial.
Patients receiving eflornithine were 52% less likely to experience disease progression, relapse, a secondary cancer or death compared to control patients. Eflornithine patients were also 68% less likely to die when mortality was considered a standalone outcome.
Neuroblastoma Australia CEO Lucy Jones thanked the federal government for making it easier for patients with aggressive cancers to access the treatment.
“On behalf of all families of children impacted by neuroblastoma, we welcome the TGA’s decision to approve eflornithine. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families,” Ms Jones said.
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Gus Rudolph, General Manager at Norgine Australia, said the company would continue to work with stakeholders to bring eflornithine to patients as quickly as possible.
“This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval.”
The newly approved treatment inhibits ornithine decarboxylase, a key enzyme involved in the synthesis of polyamines. Stopping polyamine synthesis has flow on effects that results in decreased expression of oncogenic drivers.
Eflornithine is associated with side effects such as anaemia, low neutrophils and platelets, increased liver enzymes, hearing loss or balance problems. Full blood counts, liver function tests and baseline hearing assessments are recommended prior to commencing treatment.
Around 50 children – predominantly under the age of five – in Australia are diagnosed with neuroblastoma each year, with one in two of these cases being graded as high-risk.
