The Australian Therapeutic Goods Administration (TGA) has approved IFINWIL® (eflornithine) for the treatment of adults and pediatric patients with high-risk neuroblastoma (HRNB) who have responded to prior multiagent, multimodality therapy. This registration represents a significant advancement in the treatment landscape for this rare but aggressive childhood cancer.
Neuroblastoma Australia's CEO Lucy Jones welcomed the approval, stating, "On behalf of all families of children impacted by neuroblastoma, we welcome the TGA's decision to approve IFINWIL®. 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."
Understanding High-Risk Neuroblastoma
High-risk neuroblastoma is a rare and aggressive form of cancer that predominantly affects children, with patients diagnosed at an average age of just 2 years. The disease originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumor in the adrenal glands. What makes neuroblastoma particularly challenging is its tendency to spread to other parts of the body, with metastasis often already present at the time of diagnosis.
Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases classified as high-risk. The average survival rate for children with HRNB is only 50%, highlighting the urgent need for new treatment options.
IFINWIL's Mechanism and Clinical Significance
IFINWIL® works by blocking an enzyme called ornithine decarboxylase (ODC), which is responsible for producing polyamines that play a crucial role in tumor growth and development. The drug has been investigated specifically as a post-maintenance treatment for high-risk neuroblastoma in pediatric patients with no active disease after first-line multiagent, multimodality therapy.
This approval is particularly significant because children diagnosed with HRNB typically undergo an intense treatment regimen that still leaves them vulnerable to relapse and death. Despite some improvements in survival rates, children with high-risk neuroblastoma still face a 30% chance of recurrence within the first 5 years post-maintenance, with extremely poor prognosis after relapse—as low as 15% five-year survival rate.
"Avoiding relapse is key to long-term survival," noted Dr. David Gillen, Chief Medical Officer at Norgine. "Until now, there have been no approved therapies for the post-maintenance treatment period in major markets outside of the United States."
Project Orbis Facilitates International Approval
The TGA approval came through Project Orbis, an initiative of the US FDA Oncology Center of Excellence that provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities.
"This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world," said Dr. Gillen. "By working collaboratively with international regulatory partners, we are able to help bring promising therapies to pediatric patients sooner – a goal that is especially important when time is critical."
In April 2024, Norgine submitted applications for approval of eflornithine in high-risk neuroblastoma via Project Orbis in Australia, Switzerland, and the United Kingdom, supporting the company's efforts to expand access to this treatment option in pediatric oncology.
Path to Patient Access
While TGA registration marks an important milestone, Gus Rudolph, General Manager of Norgine Australia, emphasized that the company will "continue to engage with the relevant stakeholders to bring IFINWIL® to patients as quickly as possible."
It's worth noting that IFINWIL® is not currently included on the Pharmaceutical Benefits Scheme (PBS), which would be necessary for broader patient access and affordability in Australia.
Safety Considerations
Healthcare providers and patients should be aware of potential side effects associated with IFINWIL®. According to the Consumer Medicines Information, these include risks of low red blood cells (anemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increased liver enzymes, and hearing loss or balance problems.
Industry Perspective
Norgine, a European specialist pharmaceutical company with a century-long history of bringing transformative medicines to patients, views this approval as aligned with their mission. The company has a direct presence in 16 European countries, as well as Australia and New Zealand, and helped more than 25 million patients worldwide in 2022.
For families affected by high-risk neuroblastoma, this approval represents hope for improved outcomes in a disease that has seen limited therapeutic advances. As Lucy Jones from Neuroblastoma Australia noted, they "look forward to continued support for children with aggressive cancers" as the treatment landscape evolves.
