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.
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.
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