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Orphazyme Teeters On The Brink As Arimoclomol EU Filing Is Pulled

 
• By Kevin Grogan

Danish biotech Orphazyme has withdrawn the EU marketing application for its Niemann-Pick disease type C therapy arimoclomol and all hopes for the drug’s survival now rest on a meeting with the US Food and Drug Administration.

abyss
Orphazyme’s NPC drug is on the edge of an abyss • Source: Alamy

Orphazyme’s annus horribilis is getting more horrible by the week and the latest setback has seen the beleaguered Danish biotech pull the filing in Europe for arimoclomol, its investigational treatment for the ultra-rare disorder Niemann-Pick disease type C (NPC).

The company announced on 22 March that it had withdrawn its marketing authorization application (MAA) to the European Medicines Agency for arimoclomol, a move which comes as no surprise, given that the company was informed last month by the agency's

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