Norgine welcomes PBS listing in Australia of IFINWIL® (eflornithine) for patients with high-risk neuroblastoma (HRNB)

This news is for Australian Media only

• Eflornithine is now reimbursed by the government under Australia's Pharmaceutical Benefits Scheme (PBS). The Pharmaceutical Benefits Advisory Committee (PBAC) recommended the listing of eflornithine for post-maintenance treatment to prevent relapse in patients with high-risk neuroblastoma (HRNB) who are in remission after receiving multiagent, multimodality therapy.¹
• Approximately 50 children in Australia are diagnosed with neuroblastoma each year, with nearly half classified as having high-risk neuroblastoma (HRNB). ³ These children are, on average, just 2 years old, and the average survival rate for those with HRNB is 50%.²

SYDNEY, Aug. 1, 2025 /PRNewswire/ -- Norgine, announced today the inclusion of IFINWIL^® (eflornithine) on Australia's Pharmaceutical Benefits Scheme (PBS). This listing comes just 3 months after receiving registration by the Therapeutic Goods Administration (TGA) and represents a pivotal step in improving access to essential treatments for patients with high-risk neuroblastoma (HRNB) who are in remission after receiving multiagent, multimodality therapy.

Lucy Jones, CEO Neuroblastoma Australia, welcomed the news: "On behalf of all families of children impacted by neuroblastoma, we welcome the PBS listing of IFINWIL^®. We urgently need treatments for children diagnosed with neuroblastoma and this listing ensures every child in Australia has equal access to a new therapy for this aggressive and complex cancer. Providing options to new treatments is critical in ensuring every child diagnosed has the best chance of survival."

"We thank the Federal Government for their support in ensuring early access to this treatment prior to TGA approval and PBS listing, which enabled children with high-risk disease to access the treatment within a time critical treatment window. We look forward to continued support for children with aggressive cancers."

HRNB is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life.² Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high-risk neuroblastoma.³ Neuroblastoma originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands.⁴ It is considered an aggressive tumour because it often spreads to other parts of the body (metastasises). In most cases, it has spread by the time it is diagnosed.⁴

"Eflornithine (DFMO) is a new treatment for high-risk neuroblastoma, an aggressive cancer seen mainly in infants and young children, said Dr. Toby Trahair, Paediatric Oncologist at the Sydney Children's Hospital. Having access to eflornithine (DFMO) provides clinicians with a much-needed therapeutic option. It's great to see that this new oral medication will be available to all Australian families through the PBS."

"The PBS listing of eflornithine is a landmark moment for families affected by high-risk neuroblastoma in Australia," said Gus Rudolph, General Manager, Australia at Norgine. "This decision reflects the growing recognition of the urgent need for new treatment options in paediatric oncology. High-risk neuroblastoma has limited therapeutic pathways and eflornithine offers a new approach. We are grateful to the Australian Government for providing clinicians with access to this innovative therapy and remain committed to advancing solutions in areas of high unmet medical need."

Please refer to the IFINWIL^® Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing.

Parents should speak to their doctor for guidance on the best treatment approach for their child.

Additional Information about IFINWIL^® (eflornithine)

PBS Information. Authority required. Section 100 Highly Specialised Drugs Program. Restrictions Apply. Please refer to the PBS schedule for more details.

Indication: For the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB) who have responded to prior multiagent, multimodality therapy.

Contraindications: Hypersensitivity to eflornithine or to any of the excipients.

Special Warnings and Precautions: myelosuppression, hepatotoxicity, hearing loss / hypoacusis, use in renal impairment, elderly, paediatric patients.

Interactions: No in-vivo drug interaction studies have been performed with other medicinal products.

▼This medicine is subject to additional monitoring due to approval of an extension of indications. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. You can report side effects to your doctor, or directly at www.tga.gov.au/reporting-problems.

Dr. Toby Trahair has served on advisory boards and participated in symposia sponsored by Norgine for which compensation was received. In relation to this Norgine media announcement, no compensation was provided to Dr. Trahair, and the opinions expressed are his own. Dr. Trahair has been briefed by Norgine on the approved use of this product.

NOTES TO EDITORS

About IFINWIL^®IFINWIL^® has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients after first line multiagent, multimodality therapy.⁵ IFINWIL^® is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development.⁶

For more information on IFINWIL^®, find the Public Summary Document here: Pharmaceutical Benefits Scheme (PBS) | Eflornithine; Tablet 192 mg (as hydrochloride); Ifinwil^®  or speak to your healthcare practitioner.

High-Risk Neuroblastoma (HRNB) Treatment BackgroundChildren diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.⁷ Although there have been some improvements in survival, the prognosis for children with high risk neuroblastoma is still poor with around 80% of high risk relapses occuring within 2 years from diagnosis, and long-term survival remains low, with only about 15% surviving more than 5 years post-relapse.^7,8,9 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States.¹⁰

About NorgineNorgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021.

Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world.

NORGINE and the sail logo are trademarks of the Norgine group of companies.

References

^1.  Australian Public Summary Document: Pharmaceutical Benefits Scheme (PBS) | Eflornithine; Tablet 192 mg (as hydrochloride); Ifinwil^® . Accessed July 2025^2.  https://www.neuroblastoma.org.au/about-key-statistics. Accessed July 2025^3.  ANZCHOG-Position-Statement-Eflornithine-DFMO-use-for-patients-with-high-risk-neuroblastoma-in-Australia-and-New-Zealand-v2.0-23.08.2024.pdf. Accessed July 2025^4.  https://rarediseases.info.nih.gov/diseases/7185/neuroblastoma. Accessed July 2025^5.  Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov. Accessed July 2025^6.  Meyskens FL Jr, Gerner EW. Development of Difluoromethylornithine (DFMO) as a Chemoprevention Agent. Clin Cancer Res. 1999 May;5(5):945-51^7.  Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57.^8.  Desai A, et al. Outcomes Following GD2-Directed Post consolidation Therapy for Neuroblastoma After Cessation of Random Assignment on ANBL0032: A Report From the Children's Oncology Group. J Clin Oncol. 2022 Jul; JCO2102478.^9.  London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23.^10.  FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA. Accessed July 2025

Job Number: AU-ONC-IFW-2500008Date of preparation: July 2025