Zevra’s Arimoclomol Brings Efficacy Questions To First Genetic Metabolic Diseases Adcomm

 
• By Sue Sutter
  
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The US FDA seeks input on the validity of the clinical outcome scale used in the sole pivotal trial, whether treatment benefit can be ascertained, and the robustness of the confirmatory evidence in Niemann-Pick disease type C.

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The FDA's external advisors will consider a package of confirmatory trial evidence for arimoclomol. • Source: Shutterstock

The US Food and Drug Administration's Genetic Metabolic Diseases Advisory Committee will face a host of efficacy issues with Zevra Denmark A/S’ arimoclomol at its inaugural meeting, including uncertainty about the treatment effect in the lone pivotal trial, challenges interpreting the primary endpoint, and adequacy of the confirmatory evidence.

Key Takeaways

  • The FDA questioned the validity of the clinical outcome measure, as well as whether a treatment benefit can be ascertained, in the lone randomized, placebo-controlled study of arimoclomol in Niemann-Pick disease type C.

Zevra is seeking approval of arimoclomol for treatment of Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder without...

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