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

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.