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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

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...

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Complete Response Letter Trio Raises Prospect Of Tidal Shift In Regenerative Medicine Regulation

 
• By Bridget Silverman

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US FDA’s Second Half 2025 User Fee Goals Swing Toward Respiratory Disease

 
• By Bridget Silverman

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Slowdown: US FDA’s 2025 Novel Approvals Are On Below-Average Pace

 
• By Bridget Silverman

US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 
• By Bridget Silverman

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

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US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 
• By Bridget Silverman

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

America Next: Most Novel Agents With July Goal Dates Have Been Approved Overseas

 
• By Bridget Silverman

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US.

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.