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Orphazyme's Arimoclomol Disappoints For Muscle-Wasting Disorder

Pivotal Trial Failure For ‘Pipeline-In-A-Product’

 
• By Kevin Grogan

The Danish biotech's stock sank after arimoclomol failed in a Phase II/III trial for inclusion body myositis. Orphazyme is hoping for much better news in the coming weeks as the US FDA makes its decision on the drug as a treatment for Niemann-Pick disease Type C.

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Denmark's Orphazyme reflects on disappointing data • Source: Shutterstock

As Orphazyme A/S waits to hear whether US regulators will approve arimoclomol for Niemann-Pick disease Type C, the Danish company's potential pipeline-in-a-product has failed in a pivotal trial for inclusion body myositis (IBM), a rare muscle-wasting disease.

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