Swissmedic Grants Approval for IFINWIL® to Combat High-Risk Neuroblastoma in Children

Swissmedic Approves IFINWIL® for High-Risk Neuroblastoma in Children

On July 8, 2025, Norgine, a prominent European pharmaceuticals company, announced a significant milestone in pediatric oncology: Swissmedic has approved IFINWIL® (eflornithine) as a treatment option for children aged one and older diagnosed with high-risk neuroblastoma (HRNB). This landmark decision is anticipated to enhance treatment accessibility for young patients grappling with this aggressive form of cancer.

High-risk neuroblastoma is a challenging condition, primarily affecting children under the age of five, and the average survival rate is alarmingly low at approximately 50%. The cancer develops from neuroblasts—cells that typically mature into nerve cells—and often presents as a tumor in the adrenal glands. Its aggressive nature means that by the time of diagnosis, it has often spread to other parts of the body, complicating treatment further.

Norgine's VP and Head of Global Regulatory Affairs, Jörg Plessl, expressed gratitude towards Swissmedic and the collaborators of Project Orbis, a framework established to expedite the global review and approval of innovative cancer therapies. He remarked on the unique challenges of pediatric oncology, emphasizing that swift and flexible regulatory processes are crucial given the high stakes involved in children's health.

Project Orbis allows for simultaneous submission and collaborative reviews among various international regulatory agencies, aiming to accelerate patient access to novel oncology treatments. It includes partners such as the FDA from the United States, Medicines and Healthcare Products Regulatory Agency from the UK, and the Therapeutic Goods Administration from Australia. Norgine's approval for IFINWIL® comes on the heels of similar endorsements in several regions, including the US and Australia, highlighting the company’s commitment to addressing complex disease areas with meaningful therapeutic options.

Despite the rigorous treatments currently available, the five-year survival rate for children diagnosed with high-risk neuroblastoma remains disheartening. A staggering 15% of pediatric cancer-related deaths are attributed to this condition, underscoring the urgent need for better and more effective treatments. The introduction of IFINWIL® offers hope, as it acts as a post-maintenance treatment option for patients showing no active disease after initial therapies.

IFINWIL® functions by inhibiting the enzyme ornithine decarboxylase (ODC), which is vital for polyamine synthesis—compounds that contribute to tumor growth. The lack of approved therapies in major markets outside the United States for the post-maintenance phase of HRNB treatment has heightened the anticipation surrounding IFINWIL®'s availability in Europe.

The journey towards achieving this regulatory approval has been collaborative and strategic, aiming at not merely improving access but truly enhancing outcomes for children with HRNB. The urgency is palpable, as nearly 80% of high-risk relapses occur within two years of diagnosis, and long-term survival remains grim, with only about 15% surviving beyond five years post-relapse.

Norgine has a rich history of providing transformative medical solutions for over a century, with a firm commitment to enhancing access to essential treatments. As they continue to navigate the complexities of pediatric oncology, their upcoming implementations of treatments like IFINWIL® will likely be pivotal in improving patient outcomes and survival statistics.

This approval marks a critical step not only for Norgine but for countless families facing the challenges of high-risk neuroblastoma. The collaboration with Swissmedic and other international partners, alongside their adaptive regulatory strategies, ensures progress in the field of pediatric oncology, paving the way for the development of innovative solutions to combat this aggressive cancer.