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FDA Approves IWILFIN™ (eflornithine) by US WorldMeds for Combating Aggressive Childhood Cancer

Friday, December 15, 2023

US WorldMeds (USWM, LLC) has recently gained approval from the U.S. Food and Drug Administration (FDA) for IWILFIN™ (eflornithine) 192 mg tablets, an oral maintenance therapy designed for high-risk neuroblastoma. This new therapy is intended to decrease the risk of relapse in both adult and pediatric patients who have shown at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.

Neuroblastoma, a cancer that primarily affects children under the age of 5, sees 700-800 cases diagnosed annually in the U.S., with over 50% classified as high-risk. Despite effective treatments for inducing remission, the high mortality rate in high-risk neuroblastoma is largely attributed to the risk of relapse, impacting the survival of about half of these children beyond five years from diagnosis.

Breck Jones, the Chief Executive Officer of US WorldMeds, expressed enthusiasm for the FDA approval of IWILFIN, highlighting its importance in providing a much-needed treatment option for children facing high-risk neuroblastoma. The primary goal is to prevent relapse, and IWILFIN is seen as offering new hope and improved outcomes for these vulnerable patients.

The FDA approval for IWILFIN is based on results from a multi-site, single-arm study involving children with high-risk neuroblastoma who received IWILFIN as maintenance therapy post standard care, including immunotherapy. The study demonstrated improved event-free survival (EFS) and overall survival (OS) in the IWILFIN-treated group compared to an external control group. Key findings include an 84% EFS at four years compared to 73% in the control group, indicating a 52% reduction in the risk of relapse.

IWILFIN, to be taken orally twice daily for two years, is generally well-tolerated, with manageable side effects such as hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. US WorldMeds collaborated with the Beat Childhood Cancer Research Consortium at Penn State University for preclinical and clinical research, emphasizing the importance of partnerships in advancing this therapy.

Kristen Gullo, Vice President of Development and Regulatory Affairs at US WorldMeds, expressed gratitude for the dedication of partners, particularly the Beat Childhood Cancer Research Consortium, in improving outcomes for pediatric cancer patients. The FDA approval represents hope for the high-risk neuroblastoma community and a significant step forward in the fight against this devastating disease.

IWILFIN is expected to be available in the United States in the coming weeks, offering a potential evolution in the standard of care for high-risk neuroblastoma patients. 



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