IFINWIL®: now TGA registered in Australia

Children diagnosed withneuroblastoma are on average just 2 years old2

The average survival rate of children withHRNB is only 50%2

UXBRIDGE,England,April 17,2025 -- Norgineis pleased to announce that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL® (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB),who have responded to prior multiagent,multimodality therapy.1

Neuroblastoma Australia welcomed the news today:

"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. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers." said Lucy Jones,CEO Neuroblastoma Australia.

High Risk Neuroblastoma is a rare but aggressive form of cancer,predominantly affecting children and most commonly presenting in the first 5 years of life2. Each year in Australia,approximately 50 children are diagnosed with neuroblastoma,with about half of these cases being classified as high risk neuroblastoma3,4. Neuroblastoma originates in the body's nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases,it has spread by the time it is diagnosed5.

"We are committed to improving the lives of children and their families living with high-risk neuroblastoma"said Gus Rudolph,General Manager,Norgine,Australia. "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. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL® to patients as quickly as possible."

"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. David Gillen,Chief Medical Officer,Norgine. "By working collaboratively with international regulatory partners,we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We're proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines."

Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL® is not currently included on the PBS.

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.1

Notes to Editors:

About IFINWIL®

IFINWIL® has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent,multimodality therapy.6 IFINWIL® is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines,which are important to tumour growth and development 7.

For more information onIFINWIL®,find the CMI here: IFINWIL Consumer Medicine Information (CMI)or the Therapeutic Goods Administration athttps://www.tga.gov.au/or speak to your healthcare practitioner.

High-Risk Neuroblastoma (HRNB) Treatment Background

Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapseand death.8Although there have been some improvements in survival,children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance,and have an extremely poor prognosis and low likelihood of long term survival9(e.g. estimates as low as 15% of patients will live for five years after relapsing).10 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 States11.

About Project Orbis

Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA,and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA),Australia Therapeutic Goods Administration (TGA),Canada (Health Canada),Singapore (Health Sciences Authority (HSA),Switzerland (Swissmedic),Brazil (Agência Nacional de Vigilância Sanitária (ANVISA),Israel (Ministry of Health).

In April 2024,Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB),viaProject Orbisin Australia,Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology.

About Norgine

Norgine 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

Australian Product Information,IFINWIL®: IFINWIL Product Information (PI). Accessed 16.04.25

https://www.neuroblastoma.org.au/about-key-statistics. Accessed 26.03.25

Australian Childhood Cancer Registry,Cancer Council Queensland. Australian Childhood Cancer Statistics Online (1983-2021).CCQ: Brisbane,Australia; October 2024.

ANZCHOG-Position-Statement-Eflornithine-DFMO-use-for-patients-with-high-risk-neuroblastoma-in-Australia-and-New-Zealand-v2.0-23.08.2024.pdf

https://rarediseases.info.nih.gov/diseases/7185/neuroblastoma. Accessed 16.03.25

Clinical Trial NCT02395666; Preventative trial ofdifluoromethylornithine (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.

Meyskens FL Jr,Gerner EW. Clin Cancer Res. 1999 May;5(5):945-51. 4. Sholler GLS,et al. Sci Rep. 2018 Sep 27;8(1):14445.

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.

Desai A,et al. J Clin Oncol. 2022 Jul; JCO2102478. 2. Lewis EC,et al. Int J Cancer. 2020 Dec;147(11):3152-3159

Bagatell R,DuBois SG,Naranjo A,et al. Children's Oncology Group's 2023 blueprint

LondonWB,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.

FDA approveseflornithine for adult and pediatric patients with high-risk neuroblastoma | FDAFDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA,Accessed 02.04.25