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

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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.

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

The US Food and Drug Administration appears to be looking for advisory committee input on how much regulatory flexibility should be granted to US WorldMeds, LLC application for eflornithine (DFMO) as it weighs the rare and life-threatening nature of pediatric high-risk neuroblastoma, against a new drug application based on one externally controlled study and subpar confirmatory evidence.

The Oncologic Drugs Advisory Committee will vote on 4 October on whether the sponsor provided sufficient evidence to conclude that...

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

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