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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.

  • Panelists said the treatment effect is small but clinically meaningful

Clinical data beyond the randomized, placebo-controlled study also provide reassuring confirmatory evidence of arimoclomol’s efficacy and safety, a majority of Genetic Metabolic Diseases Advisory Committee members said

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