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Uncertain Future For Orphazyme After Arimoclomol Snub In Europe

European Medicines Agency Likely To Advise Rejection Of Arimoclomol For Niemann-Picks Type C

 
• By Kevin Grogan

The EMA's medicines evaluation committee has told Orphazyme it is unlikely to recommend approval of the rare disease drug arimoclomol when it meets at the end of March, prompting the Danish firm to assess its strategic options.

mermaid
Denmark's Orphazyme is being forced to reflect on its future • Source: Alamy

The future of Orphazyme A/S is up in the air after regulators in the EU issued a strong signal that they are likely to recommend against the approval of arimoclomol, the Danish biotech's treatment for the ultra-rare disorder Niemann-Pick disease type C (NPC). This follows the stance of their US peers.

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