Norgine Advances Pediatric Oncology with Eflornithine Application to EMA

Norgine Submits Application for Eflornithine to European Medicines Agency

In a significant development for pediatric oncology, Norgine has announced the formal submission of a marketing authorisation application (MAA) for eflornithine (also known as difluoromethylornithine, DFMO) to the European Medicines Agency (EMA) specifically for the treatment of high-risk neuroblastoma (HRNB). This application highlights Norgine's dedication to expanding treatment options for children suffering from this complex and challenging cancer.

The application submission, which took place on January 7, 2025, follows previous submissions made under Project Orbis in Australia, Switzerland, and the United Kingdom in April 2024, indicating a comprehensive strategy to secure global approval for this lifesaving treatment. The eflornithine application not only opens doors for potential new therapy in HRNB but also showcases Norgine's commitment to addressing unmet medical needs in pediatric populations.

Norgine has partnered with USWM, LLC, a specialized pharmaceutical company based in Kentucky, to manage the registration and commercialization of eflornithine across Europe, Australia, and New Zealand. The collaborative effort is aimed at accelerating the delivery of this critical medication to those who need it most.

Eflornithine recently gained FDA approval as the first oral maintenance therapy for high-risk neuroblastoma, deemed effective in reducing the likelihood of relapse in both adult and pediatric patients who have undergone prior treatments. This FDA decision was informed by robust clinical trial results which demonstrated that patients receiving eflornithine experienced improved event-free and overall survival rates when compared to standard care counterparts.

Dr. David Gillen, Norgine's Chief Medical Officer, reinforced the importance of this application, stating that it marks a vital progress within the regulatory procedure for eflornithine and underscores the company's commitment to improving outcomes for patients battling HRNB, a condition fraught with significant unmet medical demands.

The CEO of Norgine, Janneke van der Kamp, echoed this sentiment, describing the EMA submission as a crucial milestone for families encountering this daunting diagnosis. She emphasized Norgine's ongoing dedication to advancing innovative therapies that specifically target the unique challenges faced by young patients and their families. The escalating risk of relapse in children diagnosed with HRNB is notably concerning, as approximately 30% of those who achieve remission after initial treatment go on to experience a relapse, significantly decreasing their chance of survival.

Understanding High-Risk Neuroblastoma

High-risk neuroblastoma is an aggressive cancer that primarily afflicts children, and it poses severe risks even after what is considered standard treatment. The clinical landscape reflects a dire need for additional maintenance therapies, particularly as many existing methods leave patients vulnerable to recurrence during the early post-treatment years, which is when the risk is highest.

Avoiding relapse is crucial for achieving long-term survival rates, but currently, there are minimal approved therapies available outside the United States aimed at sustaining remission following standard care. Eflornithine's mechanism as a post-maintenance therapy is anticipated to play a pivotal role in not only prolonging remission but also reducing the likelihood of relapse in children diagnosed with HRNB.

A Commitment to Pediatric Oncology

Norgine, a longstanding entity in the pharmaceutical realm with over 120 years of experience and annual revenues exceeding €500 million, focuses on delivering innovative healthcare solutions primarily within Western Europe, Australia, and New Zealand. The company's multi-faceted approach, which includes solid regulatory, clinical, and manufacturing capabilities, positions it to supply transformative medicines efficiently to millions of patients yearly.

In conclusion, as Norgine progresses with its regulatory efforts for eflornithine, the company remains steadfast in its mission to provide hope and transformative treatment options for families affected by high-risk neuroblastoma. This submission not only signifies a potential shift in the management of this challenging condition but also reaffirms Norgine's role as a pioneer in addressing critical healthcare needs in pediatric oncology.

Learn more about Norgine and their initiatives here.