On July 8, 2025 Norgine, a leading European specialty pharmaceutical company, reported that Swissmedic has approved the registration of IFINWIL (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB) (Press release, Norgine, JUL 8, 2025, View Source [SID1234654290]).
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"This milestone marks a further regulatory approval of IFINWIL for high-risk neuroblastoma, following approvals in the US, Israel and Australia," said Jörg Plessl, VP and Head of Global Regulatory Affairs at Norgine. "We deeply appreciate Swissmedic and the other Project Orbis partners for their keen awareness of the distinct challenges associated with paediatric oncology. Their agility in utilizing adaptive regulatory pathways is crucial because the stakes are incredibly high when it comes to children’s lives. In these situations, speed and flexibility are not just advantageous, they can make all the difference."
"By collaborating with international regulatory partners through Project Orbis, we are advancing our strategy to broaden access to innovative therapies for paediatric patients in Europe and ANZ." said Dr David Gillen, Chief Medical Officer at Norgine. "At Norgine, we’re proud to take on some of the most complex disease areas, driven by our mission to deliver meaningful treatments where they’re needed most."
HRNB is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life.2 Globally, neuroblastoma affects approximately 10.2 children per million annually under age 15, with around 50% of cases classified as high-risk at diagnosis.3,4 Neuroblastoma originates in the body’s nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands.5 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.5
Despite intensive multimodal therapy, the 5-year survival rate for HRNB remains below 50%, compared to up over 95% for infants with low-risk disease.2,6 Neuroblastoma accounts for approximately 15% of all paediatric cancer-related deaths, underscoring the urgent need for more effective treatment options.7
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.8 IFINWIL is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development.9
High-Risk Neuroblastoma (HRNB) Treatment Background
Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.10 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.11,12,13 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.14
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), via Project Orbis in Australia, Switzerland and the United Kingdom. In April 2025, the Australian Therapeutic Goods Administration (TGA) approved the registration of IFINWIL for the treatment of adults and paediatric patients with HRNB, who have responded to prior multiagent, multimodality therapy.
These regulatory milestones support Norgine’s efforts to deliver patient access to IFINWIL and bring a further treatment option in the field of paediatric oncology.