FDA Panel Will Have To Weigh Pediatric Cancer Drug’s ‘Higher Level’ Of Uncertainty Against Rare, Devastating Disease

 
• By Sarah Karlin-Smith

US WorldMeds NDA package for eflornithine for high-risk neuroblastoma provides “a higher level of uncertainty than is typically seen in oncology applications,” agency says. Though FDA seems largely satisfied with the sponsor’s externally controlled study, confirmatory evidence may be lacking. 

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FDA’s Oncologic Drugs Advisory Committee will vote 4 October on whether DFMO improves event-free survival in high-risk neuroblastoma. • Source: Shutterstock

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