Zevra’s Arimoclomol Use With Standard Of Care Complicates US FDA Adcomm Efficacy Assessment

 
• By Sue Sutter
  
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Determining the efficacy of arimoclomol alone in Niemann-Pick disease type C was challenging because most study subjects also received miglustat, an off-label drug in the US. An FDA official said panelists should assess arimoclomol’s efficacy as it was studied in the pivotal trial.

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The FDA's advisors found it difficult to untangle the efficacy of arimoclomol from miglustat. • Source: Shutterstock

Genetic Metabolic Diseases Advisory Committee members faced the difficult and unusual question of assessing an investigational drug’s efficacy when it was studied in concomitant use with an unapproved standard of care, when considering Zevra Denmark A/S’ arimoclomol for Niemann-Pick disease type C.

Key Takeaways

  • Most patients in Study NPC-002 were on miglustat therapy as part of routine clinical care, which made it difficult to separate out arimoclomol’s effects.

Ultimately, most panelists said that while they could not determine whether arimoclomol was effective on its own, the product still...

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