Miplyffa: animal efficacy model or human cells?

“Arimoclomol was shown to increase NPC1 protein expression in fibroblast cell lines from NPC patients in vitro at considerably higher concentrations than those achieved clinically, and reduced hepatic GSL accumulation and improved neurological motor ability deficit in NPC -/- mutant mice in one in vivo non-GLP study and one publication.”

“Given the limitations of the data presented in the Applicant’s resubmission, the data that support the effects of arimoclomol when used alone in patient-derived cells and in animal models of NPC are unconvincing.”