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Uncertain Future For Orphazyme After EMA Snub

Agency Set To Reject Arimoclomol For Niemann-Picks Type C

 
• By Kevin Grogan

The EMA's medicines evaluation committee has told Orphazyme it will not approve 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 left to reflect on EMA's rejection • Source: Alamy

The future of Orphazyme A/S is up in the air after regulators in the EU followed the stance of their US peers in rejecting arimoclomol, the Danish biotech's treatment for the ultra-rare disorder Niemann-Pick disease type C (NPC).

Orphazyme has been in close contact with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency since last October when it asked for more time...

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