The US Food and Drug Administration (FDA) has approved US WorldMeds’s iwilfin (eflornithine) 192mg tablets for use as an oral maintenance treatment for high-risk neuroblastoma.
The treatment cuts down the risk of relapse in adult and paediatric patients.
Individuals who have had a partial response to previous multimodality, multi-agent therapy, including anti-GD2 immunotherapy, are eligible to receive iwilfin.
To be taken orally twice a day for two years, the tablets will be made available in the US in early 2024.
The latest FDA approval is based on data from a single-arm, multi-site, externally controlled trial of paediatric high-risk neuroblastoma patients.
According to the study findings, adding iwilfin as maintenance therapy enhanced event-free survival (EFS) and overall survival (OS).
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EFS in the iwilfin arm was 84% versus 73% in the external control group four years after immunotherapy.
OS rates were 96% in subjects who received iwilfin versus 84% in the external control arm.
In further analyses to validate these results of the externally controlled study design, the decline in relapse risk was reported to be between 57% and 41%, and mortality risk between 71% and 55%.
Iwilfin is well-tolerated, with manageable side effects. Hearing loss, pyrexia, otitis media, pneumonia and diarrhoea were the most frequently seen.
US WorldMeds CEO Breck Jones stated: “We are thrilled to announce the FDA approval of iwilfin, which provides a new and much-needed treatment option for children with high-risk neuroblastoma.
“The goal for treating these young patients is to prevent relapse, and advancing therapeutic options is critical to this mission. Iwilfin offers new hope and improved outcomes for these vulnerable children.”
The company collaborated with Penn State University’s Beat Childhood Cancer Research Consortium for the preclinical and clinical research of the maintenance therapy.
