Norgine Applies for EMA Approval of Eflornithine in High-Risk Neuroblastoma

LONDON, Jan. 7, 2025 – Norgine has announced the completion of its marketing authorisation application for eflornithine, a treatment for high-risk neuroblastoma (HRNB), to the European Medicines Agency (EMA). This development follows previous submissions in April 2024 through Project Orbis in Australia, Switzerland, and the United Kingdom. Eflornithine, also known as DFMO, is aimed at addressing the needs in paediatric oncology by offering a potential new treatment for HRNB.

Norgine, in collaboration with USWM, LLC (operating as US WorldMeds), holds an exclusive licensing agreement to register and market eflornithine across Europe, Australia, and New Zealand. The US Food and Drug Administration (FDA) had approved eflornithine on December 13, 2023, as the first oral maintenance therapy for HRNB, intended to reduce relapse risk in both adult and paediatric patients who have undergone certain previous treatments. This decision was influenced by results from a clinical trial that showed enhanced event-free and overall survival rates in patients treated with eflornithine compared to those receiving standard care.

Dr. David Gillen, Chief Medical Officer at Norgine, highlighted the significance of the submission through the EU Centralised Procedure. He emphasized Norgine's dedication to expanding treatment options for HRNB, a condition with significant unmet medical needs. Janneke van der Kamp, CEO of Norgine, described the EMA submission as a crucial step for patients battling this challenging cancer. She reiterated the company's commitment to pursuing innovative therapies that meet the needs of young patients and their families, bringing hope to those affected.

Background information on HRNB indicates that children diagnosed with this aggressive form of cancer typically undergo an intense standard of care regimen. Despite this, they remain at risk of relapse and mortality, with the highest threat occurring within two years post-treatment. Approximately 30% of patients who achieve remission after initial therapy will relapse, and once this happens, the survival rate drastically drops to under 10% over four years. Therefore, preventing relapse is essential for long-term survival. Eflornithine, as a post-maintenance therapy, has shown promise in extending remission and reducing relapse risk in HRNB patients.

Norgine is a distinctive specialty pharmaceutical and consumer healthcare company with a significant annual revenue exceeding €500 million. With a history spanning 120 years, the company has consistently delivered life-changing products to patients across Western Europe, Australia, and New Zealand. Norgine's integrated approach, which includes strong commercial capabilities, profound medical, regulatory, and clinical expertise, as well as in-house manufacturing and robust supply networks, allows them to deliver high-quality medicines efficiently to over 25 million patients each year. The company prides itself on its innovative and high-performing nature, driven by a focus on operational excellence. This dedication is poised to secure its legacy of over a century of innovation and commitment to patient care, as it ventures into new therapeutic areas.