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Orphazyme Steams Ahead With Registration in Niemann Pick, Despite Endpoint Misses

 
• By Jo Shorthouse

Even with numerous endpoint misses, such is the demand for treatment options for the ultra-rare disease Niemann Pick, Orphazyme is pushing forward with its US and European regulatory filings of arimiclomol, which has no approved therapies in the US and one in the EU.

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Orphazyme Misses The Mark But Files Anyway • Source: Shutterstock

Danish rare disease company Orphazyme AS is preparing to file its lead candidate arimoclomol for Niemann Pick disease type c (NPc) with the regulators based on data from its Phase III study, despite it not showing significance on primary or secondary endpoints.

The 12-month study, which recruited 50 patients aged 2-18, was a double-blinded, placebo-controlled interventional study that aimed to show efficacy and safety of

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