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Zevra’s Arimoclomol US FDA Panel Nod Includes Nonclinical Data Doubts

 
• By Sue Sutter

The pivotal trial results, along with other data, support a conclusion of efficacy for Niemann-Pick disease type C, but nonclinical data are weak and cannot serve as confirmatory evidence for a single trial, the advisory committee majority said.

Confirm
Zevra's nonclinical data cannot provide confirmatory evidence. • Source: Shutterstock

Zevra Denmark A/S’ arimoclomol demonstrated a small but clinically meaningful treatment effect for Niemann-Pick disease type C (NPC) in the drug’s lone pivotal trial, a US Food and Drug Administration advisory committee said 2 August.

Key Takeaways

  • The advisory committee majority voted that the pivotal trial results and other clinical data supported arimoclomol’s efficacy in NPC.

Clinical data beyond the randomized, placebo-controlled study also provide reassuring confirmatory evidence of arimoclomol’s efficacy and safety, a majority of...

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

 
• By Bridget Silverman

The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year.

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.

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

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