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US WorldMeds’ Eflornithine Gets US FDA Panel Nod For Pediatric Neuroblastoma

 
• By Sue Sutter

In 14-6 vote, advisory committee said use of external control comparison to contextualize single-arm trial data demonstrated efficacy and reliance on preclinical data as confirmatory evidence was justified.  

Boy looking out window
ODAC looked outside to an external control group for a demonstration of DFMO's efficacy in pediatric neuroblastoma. • Source: Shutterstock

A majority of US Food and Drug Administration advisory committee members on 4 October voted that US WorldMeds, LLC provided sufficient evidence to conclude that eflornithine (DFMO) improves event-free survival in pediatric patients with high-risk neuroblastoma despite a nontraditional data package.

In a 14-6 vote by the Oncologic Drugs Advisory Committee, panelists in the majority said they found the sponsor’s use of an external control group to contextualize data from a single-arm study of DFMO demonstrated efficacy, and multiple sensitivity analyses conducted by the

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Maybe In May: US FDA Calls Coming On Moderna Next-Gen COVID-19 Vaccine, AbbVie’s Big ADC Bet

 
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• By Bridget Silverman

Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.

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