Major shareholder announcement
Jan 30, 2019
Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, hereby announces the receipt of notification pursuant to Section 38 of the Danish Capital Markets Act from Novo Holdings A/S on behalf of Novo Nordisk Fonden that Novo Holdings A/S as of today’s date holds a total of 2,984,368 shares in the Company, corresponding to 14.97% of the share capital and the voting rights in the Company.
Major shareholder announcement
Read moreOrphazyme 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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