Norgine, a specialty pharmaceutical company, has announced the completion of its marketing authorization application (MAA) to the European Medicines Agency (EMA) for eflornithine (difluoromethylornithine or DFMO) in high-risk neuroblastoma (HRNB). This regulatory submission aims to provide a new treatment option for patients in Europe facing this aggressive pediatric cancer.
The application to the EMA follows earlier submissions in April 2024 to regulatory authorities in Australia, Switzerland, and the United Kingdom, all under the Project Orbis initiative, which facilitates concurrent review of cancer drugs across multiple countries.
Clinical Basis for Application
The U.S. Food and Drug Administration (FDA) approved eflornithine in December 2023 as the first oral maintenance therapy for HRNB. It is indicated to reduce the risk of relapse in adult and pediatric patients who have received prior therapies. The FDA's decision was based on data from Study 3b (NCT02395666), which compared outcomes of patients treated with eflornithine to a clinical-trial-derived external control arm from Study ANBL0032 (NCT00026312).
The results of the study indicated that patients treated with eflornithine demonstrated improved event-free survival (EFS) and overall survival (OS) compared to those who received standard of care (SoC) alone. Specifically, a propensity score-matched analysis showed that eflornithine, when administered post-immunotherapy, led to significant improvements in both EFS (HR, 0.496; 95% CI, 0.294-0.835; P = .0083) and OS (HR, 0.376; 95% CI, 0.185-0.764; P = .0069).
High-Risk Neuroblastoma: An Unmet Need
High-risk neuroblastoma is an aggressive cancer that primarily affects young children. Despite intensive standard of care treatments, including chemotherapy, surgery, and radiation, approximately 30% of patients who achieve remission will relapse. Once relapse occurs, the 4-year overall survival rate drops to under 10%, underscoring the urgent need for therapies to sustain remission.
Dr. David Gillen, Chief Medical Officer at Norgine, emphasized the significance of this submission, stating, "This submission via the EU Centralised Procedure represents another important step in the regulatory process for eflornithine and further emphasises Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need."
Norgine's Commitment
Norgine has an exclusive licensing agreement with USWM, LLC (dba US WorldMeds) for the registration and commercialization of eflornithine in Europe, Australia, and New Zealand. Janneke van der Kamp, CEO of Norgine, highlighted the company's dedication, saying, "Submitting this marketing authorisation to the EMA marks a pivotal step for patients facing this challenging cancer. We are committed to advancing innovative therapies that address the unmet needs of young patients and their families, and this milestone brings us closer to offering hope where it's most needed".
