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

 
• By Sue Sutter

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.

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

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