Orphazyme reports positive results from full data set of Phase II/III arimoclomol trial in Niemann-Pick disease Type C (NPC)
Jan 30, 2019
Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced the full data set for its Phase II/III clinical trial of arimoclomol in NPC, a devastating rare genetic disorder. Treatment with arimoclomol adjunct to routine clinical care resulted in a 74% reduction in disease progression (p-value =0.0506) as measured by the primary endpoint, 5-domain NPC Clinical Severity Scale (NPC-CSS). In the predefined subgroup of patients of 4 years and older (44 out of 50 randomized patients in the trial), the treatment difference was statistically significant with a minimal disease progression at month 12 in the arimoclomol-treated group (p-value =0.0219). A highly statistically significant treatment difference was observed in another predefined subgroup analysis, requested by the European Medicines Agency (EMA), that compared arimoclomol to placebo control in patients receiving miglustat as a part of routine clinical care (p-value =0.0071).
Orphazyme reports positive results from full data set
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